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Edited Transcript of ISEE.OQ earnings conference call or presentation 5-Aug-20 12:00pm GMT – Yahoo Finance

Princeton Aug 6, 2020 (Thomson StreetEvents) -- Edited Transcript of Iveric Bio Inc earnings conference call or presentation Wednesday, August 5, 2020 at 12:00:00pm GMT

* David F. Carroll

IVERIC bio, Inc. - Senior VP, CFO & Treasurer

* Glenn P. Sblendorio

IVERIC bio, Inc. - CEO, President & Director

IVERIC bio, Inc. - VP of IR & Corporate Communications

* Kourous A. Rezaei

IVERIC bio, Inc. - Senior VP & Chief Medical Officer

* Pravin U. Dugel

IVERIC bio, Inc. - Executive VP and Chief Strategy & Business Officer

Wedbush Securities Inc., Research Division - MD & Head of Healthcare of Equity Research

Good day, and welcome to the IVERIC bio Second Quarter 2020 Results Conference Call. Today's conference is being recorded. At this time, I would like to turn the conference over to Kathy Galante. Please go ahead.

Kathy Galante, IVERIC bio, Inc. - VP of IR & Corporate Communications [2]

Good morning, and welcome to IVERIC bio's conference call. Representing IVERIC bio today are Mr. Glenn Sblendorio, Chief Executive Officer and President; Dr. David Guyer, Executive Chairman; Mr. Dave Carroll, Chief Financial Officer; Dr. Pravin Dugel, Chief Strategy and Business Officer; Dr. Kourous Rezaei, Chief Medical Officer; Dr. Abraham Scaria, Chief Scientific Officer; and Mr. Keith Westby, Chief Operating Officer.

I would like to remind you that today, we will be making statements relating to IVERIC bio's future expectations regarding operational, financial and research and development matters, including statements regarding the impact of the COVID-19 pandemic on our research and development programs, operations and financial position and on the practices of retinal physicians and the conduct of clinical trials, our expectations to use GATHER1, our previously announced clinical trial for Zimura for the treatment of geographic atrophy as a Phase III clinical trial, our development and regulatory strategy for Zimura and our other product candidates, including our expectations for a second Phase III clinical trial, GATHER2, evaluating Zimura for the treatment of geographic atrophy and our expectations of our Phase IIb screening trial evaluating Zimura for the treatment of autosomal recessive Stargardt disease, our hypothesis regarding complement and the HtrA1 inhibition as a mechanism of action for the treatment of geographic atrophy and potentially other retinal diseases.

Our projected use of cash and cash balances, the timing, progress and results of clinical trials and other research and development activities and regulatory submissions, the potentiality and development potential of our product candidates, the size of the potential market for indications our product candidates are intended to treat and the potential of our business development strategy.

These statements constitute forward-looking statements for the purposes of the safe harbor provision under the Private Securities Litigation Reform Act of 1995. These statements cover many events and matters that are subject to various risks that could cause actual results to differ materially from those expressed in any forward-looking statements, including risks relating to the future progression of the COVID-19 pandemic and its impact on our research and development program, operations and financial position, initiation of the progress of research and development programs and clinical trials; availability of data from these programs; reliance on contract development and manufacturing organization, university collaborators and other third parties; establishment of manufacturing capabilities; expectations for regulatory matters; need for additional financing and negotiation and consummation of business development transactions and other risks.

I refer you to our SEC filings and in particular to the risk factors included in our current report on Form 8-K filed on June 17, 2020, for a detailed description of the risk factors affecting our business. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we disclaim any obligation to do so, except as required by law.

I will now turn the call over to Glenn.


Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [3]


Thanks, Kathy, and good morning, everyone. We appreciate and thank you for joining our call this morning. First, I hope you and your families are safe and healthy as we continue to navigate through these very challenging times. Here at IVERIC, we are very pleased with the level of execution we have achieved during the second quarter. We are thrilled to have reached another major milestone with Zimura.

In June, we announced positive 18-month results from GATHER1. Our first Phase III clinical trial for Zimura, a novel complement C5 inhibitor for the treatment of geographic atrophy, or GA, secondary to age-related macular degeneration or AMD. The GATHER acronym for Zimura Phase III clinical trials represents GA therapy. The 18-month results from GATHER1 indicated continuous Zimura treatment benefit with a favorable safety profile in patients with GA secondary to AMD. We think this is an impressive achievement since we believe GATHER1 is currently the only Phase III clinical trial showing suppression of GA growth with continuous treatment for 18 months.

Shortly following the positive results from GATHER1, we initiated patient enrollment in GATHER2, our second Phase III clinical trial for Zimura for the treatment of GA secondary to AMD. Our main priority is to aggressively drive patient recruitment and retention in the GATHER2 clinical trial. If the primary endpoint is achieved at month 12, we intend to file for approval of Zimura with the U.S. Food and Drug Administration and the European Medicines Agency.

The initiation of patient enrollment in GATHER2 brings us another step closer to potentially delivering a clinical meaningful therapy safely to patients with GA, where there is currently no treatment. We continue to work closely with the FDA, and we are pleased to receive FDA fast-track designation for Zimura for the treatment of GA secondary to AMD. In addition to the complement systems' potential role in GA and Stargardt disease, we believe, based on scientific data, that Zimura may have potentially -- may have a potentially impactful role in treating intermediate AMD as well as wet AMD. We believe there is strong scientific rationale to support the development of Zimura in multiple forms of AMD early and advanced, dry and wet.

We believe HtrA1, or high-temperature requirement A serine peptidase protein 1, could be another important target in the treatment of AMD. We called this program IC-500. HtrA1 is a small molecule inhibitor that is a promising compound in our pipeline. HtrA1 up-regulation has been implicated as a strong risk factor in the development of dry AMD. We are particularly encouraged by IC-500's ability to engage the target, both extra and intracellularly in early preclinical work.

Based on current time lines, we are planning to submit an IND to the U.S. FDA for IC-500 in GA secondary to AMD in 2021. Although bringing to more, to patients is our top priority, we continue to focus on our gene therapy program in orphan inherited retinal diseases. Kourous will review the details of our gene therapy pipeline in a few moments.

Following the positive GATHER1 18-month data, we strengthened our balance sheet with an underwritten public offering and a concurrent private placement with Vivo Capital and Samsara BioCapital, raising approximately $160 million in gross proceeds. Dave will cover our cash runway later in this call. We believe this fundraising enables us to further execute on our strategy to develop and deliver retinal treatments through our Zimura -- through Zimura, our gene therapy programs and IC-500 with potential to create long-term shareholder value.

At the beginning of the second quarter, we welcomed Dr. Pravin Dugel, who many of you know, to IVERIC bio. Pravin is a globally recognized retinal specialist, who has an extensive network and long-standing relationships with the bio and pharma ophthalmic industry. As our Executive Vice President and Chief Strategy and Business Officer, Pravin's experience and network will be instrumental in helping us build alliances with potential future collaborators, investors and other stakeholders. Pravin is helping to lead the company's strategy as we advance our portfolio of therapeutics and gene therapy R&D programs targeting multiple retinal diseases.

In July, we had the privilege of announcing the addition of Dr. Mark Blumenkranz to our Board of Directors. Mark is a biotech industry leader and internationally known vitreoretinal specialist, with a notable expertise in pharmaceuticals for age-related macular degeneration and ocular gene therapy. Mark has cofounded multiple biotech and medical technology companies. His experience and expertise in leading and building biotech companies, this further strengthens our Board. I'd now like to turn the call over to Pravin.


Pravin U. Dugel, IVERIC bio, Inc. - Executive VP and Chief Strategy & Business Officer [4]


Good morning, everyone. I hope you're all well. Thank you, Glenn, for the kind introduction. And this is an exciting time at the company. And it is my great pleasure to work closely with Glenn, David, Kourous and all my new colleagues. We are very excited by these 18-month GATHER1 results. This is a significant milestone for Zimura and potentially a significant advancement for patients with GA secondary to AMD.

We believe the 18-month data that we reported this past June further validates our 12-month results regarding Zimura's continuous positive treatment effect with a favorable safety profile in GA secondary to AMD and highlights the potential role of complement C5 inhibition in this disease. In the GATHER1 clinical trial, Zimura met its prespecified primary efficacy endpoint at 12 months and reached statistical significance in the international multicenter randomized, double-masked, sham-controlled Phase III clinical trial in GA secondary to AMD.

The reduction in the mean rate of GA growth over 12 months was 27.38% with a p-value of 0.0072 for the Zimura 2-milligram group as compared to the corresponding sham control group and 27.81% with a p-value of 0.0051 for the Zimura 4-milligram group as compared to the corresponding sham control group. These data for both dose groups were statistically significant. These positive 12-month data are further supported by the 18-month results, which we reported in June.

Over 18 months, the reduction in the mean rate of GA growth was 28.11% for the Zimura 2-milligram group as compared to its corresponding sham group and 29.97% for the Zimura 4-milligram group as compared to its corresponding sham control group. The primary efficacy endpoint was prespecified at 12 months using all of the power to detect a statistically significant difference. Therefore, the p-values at the 12-month statistical analyses are descriptive in nature. The descriptive p-value for the treatment effects at month 12 were a p-value of 0.0014 for the Zimura 2-milligram group and a p-value of 0.0021 for the Zimura 4-milligram group.

We believe having 18-month positive data with continuous treatment is a key differentiating factor for us when compared to other product candidates being developed for GA. Another key differentiating factor for this trial is that the treatment effect was observed in the very first measurement at 6 months with an increasing absolute difference between the treated group and the sham at each subsequent measurement time point. In other words, if these results are replicated in GATHER2 trials, we believe a doctor would be able to tell his or her patient that this drug has been -- has shown an effect as early as 6 months and then may have an increasing effect with every subsequent injection thereafter.

We do not believe this impactful efficacy profile has been observed in any other GA clinical trial to date. Zimura's favorable safety profile, another potential differentiating factor was maintained throughout the 18-month trial with no investigator-reported Zimura-related adverse events, no cases of endophthalmitis and no Zimura-related inflammation. The reported incidence of CNV in the untreated fellow eye was 11 patients, 3.8%; and in the study eye was 3 patients, 2.7% in the sham control group; 2 patients, 7.7% in the Zimura 1 milligram group; 8 patients, 11.9% in the Zimura 2-milligram group; and 13 patients, 15.7% in the Zimura 4-milligram group.

Note, we believe these rates of CNV were lower than what has been published for C3 inhibition, despite the fact that there were more Zimura injections administered over a longer period of time in a patient population with faster progressing disease. This is particularly the case when you look at the ratios of the incidents in the treated arms versus sham. The most frequently reported ocular adverse events in GATHER1 were related to the injection procedure and not the drug.

These GATHER1 reports are from the pre-COVID era, making the trial what we believe is the only pure pre-COVID positive Phase III clinical trial in GA. We believe there is a new environment for clinical trial execution and that the robust data from GATHER1 may help drive recruitment and retention of patients in GATHER2 and any future trials we may conduct for Zimura.

In the challenging COVID-19 pandemic era, we believe that investigators will be more enthusiastic and comfortable recruiting and retaining patients in a clinical trial with a drug that already has high-quality positive Phase III data. We recognize the challenges that retinal physicians face with their practices and conducting clinical trials in the COVID era, and we're working closely to support collaborating physicians. Kourous will discuss how we plan to leverage the quality of the GATHER1 results to maximize patient recruitment and retention for GATHER2 Phase III clinical trial.

Turning to our business development. We plan to continue our aggressive, but selective efforts as we continue to explore our options for future development and potential commercialization of Zimura, including potential out-license and collaboration opportunities.

Thank you for your time. I will now turn the call over to Kourous.


Kourous A. Rezaei, IVERIC bio, Inc. - Senior VP & Chief Medical Officer [5]


Thank you, Pravin, and good morning, everyone. We are excited to have the robust Zimura GATHER1 results presented at the association for Research in Vision and Ophthalmology, Arvo Annual Meeting by Dr. Karl Csaky, T. Boone Pickens' Senior Scientist and Director of the Molecular Ophthalmology Laboratory at the Retina Foundation of the Southwest and also presented at the American Society of Retina Specialists, ASRS Annual Meeting run 2 weeks ago by Dr. Baruch Kuppermann, Chairman of the Department of Ophthalmology at the University of California Irvine.

In the coming months, we are planning for our data to be presented by key opinion leaders at the major retina meeting in the U.S. and around the world. The positive GATHER1 clinical trial data has ignited a significant enthusiasm in our investigators to participate and enroll patients in the GATHER2 clinical trial. Further, we believe that the early onset and continuous treatment effect demonstrated in the GATHER1 clinical trial will be a key motivator for retention in the GATHER2 trial.

Our experienced clinical trial team has worked tirelessly in the past few months to support the smooth and expeditious initiation of the GATHER2 clinical trial. Some of these efforts include hosting multiple virtual websites with investigators and study coordinators in the U.S. and around the world. Frequent communications with the clinical trial site coordinators to assess and support their preparedness for COVID-19 and minimizing any potential disruption.

Making arrangements for patients to remotely perform certain trial-related tasks to minimize the time spent by the patients at the clinical trial sites and to reduce their exposure risk and ensure a safe environment for our patients. We thank all our principal investigators and their staff for their enthusiasm and support for the GATHER2 clinical trial.

Now I would like to provide some details regarding the design of the GATHER2 trial. GATHER2 is an international randomized, double-masked, sham-controlled, multicenter Phase III clinical trial, evaluating the safety and efficacy of Zimura 2-milligram in patients with geographic atrophy secondary to age-related macular degeneration. We are planning to enroll approximately 400 patients in this clinical trial.

Patients will be randomized 1:1 into 2 cohorts. The first cohort receiving monthly administration of Zimura 2-milligram for 12 months and the second cohort receiving monthly administration of sham. The prespecified primary efficacy endpoint is the mean rate of change in geographic atrophy growth over 12 months, measured by fundus autofluorescence at 3 time points; baseline, month 6 and month 12, very similar to the GATHER1 clinical trial.

If the primary efficacy endpoint is met at month 12, we are planning to file for marketing approval of Zimura for the treatment of geographic atrophy, secondary to age-related macular degeneration with the FDA and EMA. At month 12, we plan to re-randomize patients in the Zimura 2-milligram arm to receive either monthly or every-other-month administrations of Zimura 2-milligram. The final safety evaluation will be performed at month 24 for all patients.

Turning now to Stargardt disease. We currently have an ongoing Phase IIb screening clinical trial assessing the safety and efficacy of Zimura in patients with autosomal recessive Stargardt disease. Initially, we enrolled 95 patients in this trial. We have reopened the enrollment in this trial to add approximately 25 patients with the goal of enrolling a total of 120 patients as was initially intended in the protocol for this trial. We remain masked for this trial and plan to perform data analysis when all the patients have reached the month-18 time points.

Regarding our gene therapy programs, we continue with our IND-enabling activities and natural history studies for IC-100, our product candidate for rhodopsin-mediated autosomal-dominant retinitis pigmentosa and are planning to initiate the Phase I/II clinical trial for the first half of 2021. We also continue with the IND-enabling activity and natural history studies for IC-200, our product candidate for BEST1-related retinal diseases and are planning to initiate the Phase I/II clinical trial next year in 2021.

Our minigene programs continue to move forward. We are currently optimizing the minigene construct for our miniCEP290 program and plan to select the leads construct later this year. Thank you for your time. And please stay safe. I will now turn the call over to Dave. Dave?


David F. Carroll, IVERIC bio, Inc. - Senior VP, CFO & Treasurer [6]


Thank you, Kourous, and good morning, everyone. I'd like to highlight a few items from our press release this morning and also update our year-end cash guidance and our expected cash runway.

For the quarter, our net loss totaled $18.6 million or $0.32 per share compared to a net loss of $14.4 million or $0.35 per share for Q2 2019. This increase in net loss was driven primarily by an increase in R&D expenses, offset by a favorable settlement of a state income tax audit. Year-to-date, our net loss totaled $33.7 million or $0.61 per share compared to a net loss of $26.9 million or $0.65 per share for the same period in 2019, again, due to an increase in R&D expenses offset by a favorable settlement of the state tax audits.

Turning to our expected year-end cash balance and cash runway. We raised approximately $150 million in our June 2020 public offering and concurrent private placement. We now expect our year-end cash balance will range between $215 million and $220 million. We estimate that our cash will be sufficient to fund our capital expenditures and operating expenses as currently planned for at least mid-2024, excluding any potential approval or sales milestones payable to the Archimex Corp. or any potential commercialization expenses towards Zimura.

These estimates are based on our current business plan, which includes the continuation of our clinical development programs for Zimura, the progression of IC-100 and 200 gene therapy programs into the clinic and the advancement of our IC-500 development program. Our estimates assume that we will enroll approximately 400 patients for the GATHER2 trial. If facts and circumstances change, we'll adjust our guidance accordingly. Of course, these estimates do not reflect any additional expenditures resulting from the potential in-licensing or acquisition of additional product candidates or technologies or any associated development that the company may pursue.

I'll now turn the call back over to Glenn. Thank you for your time.


Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [7]


Thanks, Dave, and I appreciate that. So just to recap the second quarter, despite these very challenging times, it was a good quarter for IVERIC bio.

There's a number of key takeaways: we completed our 18-month results for GATHER1. And as you've seen and we discussed today, with continuous effect and a good safety profile; second, we raised $160 million in gross proceeds, further strengthening our balance sheet and adding to our cash run normally; and third, although it was a difficult discussion back in March when we decided to pause the GATHER2 trial, we have now begun that trial and are recruiting patients.

So a good quarter for us. So I'd like to thank all of you for listening today to our call and for your continued support. And now I'll turn the call over to the operator so that we can open up the line for any questions.


Questions and Answers


Operator [1]


(Operator Instructions)

We will now take our first question from Stacy Ku of Cowen.


Stacy Ku, Cowen and Company, LLC, Research Division - Equity Research Associate [2]


Congratulations on the progress. So first question, given the time spent for sites in GATHER2 for Phase III and the recent trial initiation, how should we be thinking about the pace of enrollment? Would you be willing to give us some guidance on timing?

And my second question is around something that was briefly touched upon during your prepared remarks, but curious if you could elaborate what the team is thinking in terms of potential ex-U. S. partnerships. When would you ramp up these conversations?


Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [3]


Thank you, Stacy. It's Glenn, and thanks for the questions. First, on the timing for GATHER2, I mentioned a couple of things. It's early days and this is also a very competitive environment. And I think you've seen our execution in the past. So while we are dealing with a unique situation here with COVID, and I think as we've updated you through the second quarter, we've kept a very active and continuous dialogue with the investigators.

So at the current time, we're not going to provide any guidance as to when we finish the trial. So -- but we will continue to move at a pace that's as quick as we can. The key to that success will be the number of sites, the quality of the sites and obviously, the patient enrollment. So I am going to provide you detailed guidance at this point and the fact that we've got a pretty experienced team doing this.

As for the second question as it relates to collaborations. I think Pravin and his prepared statements did talk about that. Again, priority one for us today is the recruitment and retention of patients in GATHER2, but we are going to continue to explore our options for potential commercialization of Zimura, including a potential our-license. As you know, we do not have operations overseas even with the successful capital raise in a small company. So partnership at the right time will be an important part of our efforts to commercialize Zimura. Thanks for the calls -- thanks for the questions, Stacy.


Operator [4]


And we will now take our next question from David Nierengarten of Wedbush.


David Matthew Nierengarten, Wedbush Securities Inc., Research Division - MD & Head of Healthcare of Equity Research [5]


I'm sorry. I was on mute. Apologies. A couple of questions from me. First off, the...


Glenn P. Sblendorio, IVERIC bio, Inc. - CEO, President & Director [6]

Originally posted here:
Edited Transcript of ISEE.OQ earnings conference call or presentation 5-Aug-20 12:00pm GMT - Yahoo Finance

Opinion: With any COVID-19 vaccine, humanity will face its biggest moral test – The Province

By John Authers

The coronavirus pandemic has stress-tested the world. Beyond challenging human fortitude, national health services and international rivalries, it has forced a series of moral choices. Many have provoked impassioned disagreement over whether governments can force businesses and schools to close, over sacrifices for the sake of the elderly and, most bitterly and surprisingly, over whether being asked to wear a simple face mask infringes individual liberty.

The toughest moral test lies ahead. The biomedical industry and research facilities around the world are progressing toward creating a vaccine that would offer the best chance to end the pandemic and return life to normal. But the moral dilemmas provoked by the development and distribution of a vaccine will drive ever deeper debates.

The issues strike at profound divisions between schools of ethics. The newly published The Ethics ofPandemics,an anthology edited by philosophy professor Meredith Schwartz of Ryerson University in Toronto, presents contrasting views of academics, doctors and commentators along with a series of impossibly difficult case studies. The scientific, economic and political choices involve moral issues that have divided ethicists for centuries:

How to develop it?

The U.S government says the Covid-19 vaccine will be developed at warp speed. But vaccines take years to develop, for good reasons, and none of the benefits can be realized if they are released before they are safe. A failed Covid-19 vaccine could even compromise confidence in other vaccinations, threatening a return of measles, polio and other plagues.

Testing shortcuts are available but fraught. The first rule of deciding when theyre justified, explains Arthur Caplan, the head of bioethics at the NYU Langone hospital system in New York, is that risks can be balanced against the prospect of better data. Thus, skipping animal testing may pass muster since the data from testing humans is better.

That leads to the issue that divides teams at Moderna Inc. in Boston and at Oxford University in England who are working on the two most promising attempts to find a vaccine. How much risk of harming humans can they justifiably take? The best way to accelerate the process could fall afoul of the long-established obligations of medical ethics, from the Hippocratic oath to do no harm.

That pledge is as old as ancient Greece, it aligns with Christian teaching, and with the powerful school of rights-based philosophy identified with the 18th-century German philosopher Immanuel Kant, which holds that people should never treat humanity as a means to an end. Whatever the ultimate positive consequences, Kantians argue, there is no right to harm anyone. Virtuous ends do not justify unethical means.

Immanuel Kant

In human challenge trials,which have been used to test cholera and dengue vaccines, volunteers are injected with a vaccine and then deliberately infected with the germ that researchers are hoping to neutralize. The subjects are tightly monitored, and results are available within weeks. Researchers at Oxford are developing strains of the coronavirus in preparation for such a trial alongside a much larger conventional study, as are the National Institutes of Health in the U.S. Such a study will require 150 volunteers at the most.

Moderna opted against human challenge trials, and instead started a conventional trial with 30,000 test subjects in July. Volunteers are given either the vaccine or a placebo, and then go about their daily lives as the pandemic rages. Moderna hopes to have scientifically reliable results by the end of the year. Tal Zaks, Modernas chief medical officer, said he expects this approach to reveal how the vaccine behaves with different groups of people and in different regions. By testing in the real world, he said, results can be superior to the outcome of challenge tests, which are held in laboratory conditions.

But the conventional approach is slower, and leaves much to chance. Oxfords attempt to hold such a study in London and Oxford earlier this year came just as the epidemic was beginning to decline in the U.K., making it hard to draw firm conclusions. A rival research team at Imperial College, London, has the same problem and is looking to hold a trial in another country.

Further, doctors are morally obliged to tell volunteers how to avoid getting infected. They cannot tell them to go maskless, or to seek out crowded spaces, even though from a narrowly scientific point of view this would improve their test results. Its also impossible to monitor so many volunteers closely enough to determine if they are reporting their experiences inaccurately and skewing the results.

Rutgers University bioethicist Nir Eyal says that coronavirus challenge testing in the U.S could simultaneously maximize utility and respect rights. Researchers would use only informed, willing, low-risk volunteers from a population that is already in high-risk areas, he said.

Volunteers are abundant. An advocacy group called 1 Day Sooner has found 32,000 volunteers in 140 countries, mostlybetween the ages of 20 and 30, (old enough to consent but much less exposed to serious harm from Covid-19 than their elders) with no relevant underlying medical conditions. Strongly believing in effective altruism, Josh Morrison, who heads 1 Day Sooner, voluntarily donated one of his kidneys to a stranger, as did others helping with the campaign.

But Kantian objections are serious. Michael Rosenblatt, a Harvard Medical School professor and former chief medical officer of Merck Inc., objects that human challenge studies should only be contemplated when some lifesaving treatment, such as an antiviral medicine, is available for a candidate who gets sick. There is no such cure for Covid-19.

Then there is the problem of the unknown. Vaccines must pass muster with libertarians, descended from figures such as the enlightenment philosopher John Locke and the founding fathers of the U.S., who build morality around individual freedom. To counter libertarian objections, researchers must obtain informed consent.

Brazilian pediatric doctor Monica Levi, one of the volunteers who received the COVID-19 vaccine, works at the Specialized Clinic in Infectious and Parasitic Diseases and Immunizations (CEDIPI), in Sao Paulo, Brazil, on July 24, 2020.NELSON ALMEIDA/AFP via Getty Images

Rosenblatt argues that when it comes to Covid-19, Its pretty hard to have informed consent when we barely know anything about this yet. There are fears that the virus can cause lasting damage even in twentysomethings, for example, but little clear evidence. Can volunteers really consent to expose themselves to such poorly understood risks?

Finally, there is the appalling possibility of a volunteer dying. In 1999, this happened to Jesse Gelsinger, a healthy 18-year-old with a rare metabolic genetic disorder who volunteered for a conventional safety trial (not a challenge trial) of a virus-based gene therapy. His death was both a personal tragedy and a scientific disaster that set the field of gene therapy back by at least two decades, Rosenblatt said. That hiatus deprived a generation of patients with genetic disorders of treatments.

Morrison, of 1 Day Sooner, defends the right to volunteer for testing. Estimates at present are that the risk of death from Covid-19 for people in their 20s with no pre-existing conditions is under one in 10,000 less than the risk of dying in childbirth while soldiers (whether volunteer or conscripted) face a far higher chance of dying on the battlefield.

How to pay for it?

A vaccine is meaningless if people are unable to afford it, said John Young, the chief management officer of Pfizer Inc. Nobody asserts that drug companies should be able to charge whatever the market can bear for a Covid-19 vaccine.

But private companies like Pfizer have a responsibility to shareholders. Moreover, anyone who develops a successful coronavirus vaccine will have performed an immense service to humanity and will deserve to be rewarded. And so Pfizer defends its right to make a profit.

Pfizer has a $2 billion deal with the U.S. government to supply as many as 600 million doses of the vaccine it is developing. Many of its competitors are in collaborations with public universities, or receive state funding. That raises an intensely ideological issue: Should a private company be free to set prices for a public good developed with government aid?

We have to make a profit out of the first product, Moderna chief executive Stephane Bancel told Yahoo Finance. We have invested $2 billion of our shareholder capital since we started the company. We need to get a return. But Moderna has also received some $955 million in government funding to finance its big test. According to the Financial Times, Moderna is planning to price its vaccine at $25-$30 per dose, significantly above the $19.50 at which Pfizer is selling each of 100 million doses to the U.S..

Meanwhile, AstraZeneca PLC says it will sell the vaccine it is developing with Oxford to European governments at no profit, while Johnson & Johnson says it will sell its vaccine at a not-for-profit price for emergency use.

A man walks past a sign at an AstraZeneca site in Macclesfield, central England May 19, 2014.REUTERS/Phil Noble/File Photo

The issue is already very political. Five pharma industry leaders have had to testify on their pricing plans before a committee of the U.S. House of Representatives, and Democratic-sponsored bills are in Congress to stop price gouging. They have some Republican support.

Representative Lloyd Doggett, a Texas Democrat who is sponsoring one such bill, told Politico that a drug companys claim that its providing a vaccine at cost should be viewed with the same skepticism as that by a used car salesperson.

Once governments have bought the vaccine, should they require patients to pay for their own shots? Most people with money would happily pay much more than $30 to free themselves from the coronavirus. But in the many developed countries with nationalized health systems, the question doesnt arise: taxpayers pay, and the vaccine is free for patients.

The U.S., however, has a political issue on its hands. Senator Patty Murray, a Washington Democrat, now backs a bill to ensure that every American has a right to a free vaccine. Meanwhile, the deal with Pfizer will result in free immunizations. Having established the principle of taxpayer-paid vaccines, it could be hard to retreat.

These are issues decided within countries. When it comes to international cooperation, poorer countries complain about vaccine nationalism. In the U.K., Prime Minister Boris Johnson pulled out of the EUs so-called Inclusive Vaccines Alliance in a move attacked for playing to his Brexit-friendly political base.

Wealthy countries have little incentive to collaborate with poor ones. Costa Rica led an effort with the World Health Organization to set up a new Covid-19 Technology Access Pool that would share research and then coordinate production and also share the vaccine once it was ready.

But the list of countries that responded is telling. The U.S., China, Canada and Japan are all absent, while the only European countries to sign up have been Belgium, Luxembourg, the Netherlands and Norway. A group of much smaller developing nations has been left to build a collaboration even though the virus knows no boundaries, and it is in all countries interest to stamp it out everywhere.

The virus originally thought to only attack the lungs ravages almost every part of the body. Stock/Getty

Meanwhile, rich countries are prospectively buying up vaccines before they have even been cleared for use. The U.S.-Pfizer vaccine deal, and a similar deal with Glaxo PLC and Sanofi AG, uses American buying power to avoid excessive prices. Britain has done four separate deals with providers for 250 million doses.

What about the poorer countries who may have to pay more for the vaccine? For now, attempts at vaccine justice have been left to philanthropies such as the Gates Foundations Vaccine Network.

How to ration it?

The pharmaceutical industry cannot produce enough vaccine for the entire global population of almost 8 billion all at once. Therefore, rationing is inevitable. Some people will have to wait. Who gets to make these decisions, and by what criteria?

Within the U.S., various medical bodies and government agencies claim authority to draw up the guidelines. No one seems empowered to adjudicate.

The principle is to protect those most likely to be harmed, said Caplan of NYU Langone. That leads to one point of clarity: Medical workers go first. Theyre obviously at risk, and have a duty to put themselves in harms way.

But after this, following his criterion leads to prioritizing some of the least privileged in society not because they are underprivileged and deserve help, but because they are most at risk.

Statistically, prisoners follow doctors and nurses on the list of people most likely to be harmed. As prisons are Covid-19 incubators, Caplan suggests that vaccinating inmates would limit the diseases spread.

Calgarians wear masks as they walk along Stephen Avenue Mall in downtown Calgary on Wednesday, July 29, 2020.Gavin Young/Postmedia

Within the U.S., Native-American communities are grievously affected, and therefore have a case for priority. The same is true of some other ethnic minorities, largely because they tend to live in crowded communities, and because higher rates of poverty make them more likely to suffer the underlying conditions that make Covid-19 more deadly.

People are also more at risk if they cannot work from home. In an email, Anthony Skelton, a philosophy professor at the University of Western Ontario and Lisa Forsberg of the Oxford Uehiro Centre for Practical Ethics, make a case for sending those in work-at-home professions to the back of the line. To the extent that racial minorities might live and/or work in conditions that make them less able to avoid coming into contact with infected individuals, the case for giving them priority over people who can work from their home office seems strong, the scholars wrote.

All of these proposals spring from prioritizing people according to risk, but might in practice look like the kind of redistributionist social-justice crusading that provokes controversy, particularly in the U.S.

Rationing could also be affected by where the vaccine was tested. In the case of AIDS, experimental treatments were assessed in Africa, where testing was cheaper, but the treatments then went to developed countries. Severely affected African countries had to pay prohibitive prices as the disease took hold.

Africa could become a Covid-19 test site if regulators do not permit human challenge tests elsewhere. If large-scale testing does happen there, justice will demand that early supplies of the vaccine are made available to Africans, even at the expense of people in the researchers home country.

How to roll it out?

Vaccinations work best when everyone receives them, since germs that cant infect people tend to wither away.

But all vaccines come with risks. That creates a free-rider problem. The best option from a self-interested point of view is that everybody else has the shot (eliminating your personal risk of catching Covid-19) but that you dont (avoiding any personal risk of side-effects). Taxes have the same problem. Taxes are compulsory. Does that mean vaccination should be compulsory, too?

The public-health case for compulsion is strong. But libertarians have a problem with forcing a potentially harmful vaccine on someone without the informed consent thats hard to procure in societies skeptical of experts and low on social trust.

How can the vaccine reach a critical mass without compulsion? Caplan suggests leaving compulsion to private entities. An employer might demand vaccination as a condition of reporting for work. A university might impose the same requirement on faculty and students. A vaccine might be dangled as a golden ticket to return to theaters, cinemas, night clubs or sports events. Governments or foundations could even pay people to receive a shot.

By this thinking, those who assert their right not to be vaccinated would be free to work from home and home-school. They would be voluntarily narrowing their own freedom of movement and assembly.

Yet societies would pay a price. The virus has divided humans in countless ways already. If many citizens opt to stay unvaccinated, the virus and the messy ethics of compelling vaccination will have helped to create another permanent division.

This column does not necessarily reflect the opinion of the editorial board or Bloomberg LP and its owners.

John Authers is a senior editor for markets. Before Bloomberg, he spent 29 years with the Financial Times, where he was head of the Lex Column and chief markets commentator. He is the author of The Fearful Rise of Markets and other books.

2020 Bloomberg L.P.

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Opinion: With any COVID-19 vaccine, humanity will face its biggest moral test - The Province

These Companies Are Seeking a Cure for Sickle Cell. And Its Just the Beginning for Some Gene Therapy Stocks. – Barron’s

Victoria Gray, a 34-year-old mother of four in Forest, Miss., had been going to the hospital seven times a year for transfusions to help with the severe pain brought on by her sickle cell disease. In July 2019, she volunteered for a radical new technology known as gene editing. She has been free of the pain and suffering since.

I chose to participate in this trial because of hopehope that it would change my life, Gray tells Barrons. And it has already in so many ways.

Her successful treatment gives hope to the 100,000 other Americans who suffer from sickle cell. It also illuminates the enormous potential for the companies that are pioneering genetic medicine to cure diseases with a one-time treatment.

Sickle cell is among their first targets. The companies include the sponsors of Victoria Grays clinical trial Crispr Therapeutics (ticker: CRSP) and Vertex Pharmaceuticals (VRTX)as well as biotechs like bluebird bio (BLUE), whose gene therapy for sickle cell is further along in testing and could be introduced by 2022.

For these companies, genetic therapies for sickle cell could fetch annual revenue of several billion dollars apiece. A recent Bank of America Securities report predicts that new treatments for sickle cell will surpass $6 billion in sales by 2028. That is meaningful for a company of any size, but particularly so for these biotechs. Crispr doesnt yet have sales. Bluebirds revenue last year was $45 million, and Vertexs, $4 billion.

More important, the sickle cell therapies will demonstrate how such genetic technology could open the door to curing dozens of other diseases.

Investors seem persuaded. They have lifted Crispr stock threefold since March to a recent high of $97, which values the development-stage company above $6 billion. Hitting a record high of $306, Vertexs value has topped $75 billion, while bluebirdwhose investors have waited nearly a decade for their paydaytrades at a more modest market cap of $4.2 billion. Biotech valuations may be hard to rationalize these days, but that reflects the radical changes that new technologies are bringing to the health-care business. One need look only at this years fourfold rise of the messenger RNA vaccine leader Moderna (MRNA).

The focus on sickle cell is a turnaround for the pharmaceutical industry, which had long ignored the inherited disorder. Sickle cell mainly afflicts Black Americans and residents of poor African nations. It is the most commonly diagnosed genetic disorder among newborn Americans. Yet it has not had as much funding as some less-common inherited conditions. Cystic fibrosis, for example, affects one-third as many Americans, but researchers at Duke University have shown that it has historically received more than seven times the federal and foundation research funding per patient.

This year, two new sickle cell drugs came on the market. And sometime next year, bluebird will ask the U.S. Food and Drug Administration to approve its sickle cell gene therapy. Crispr and Vertex hope that they will not be not far behind.

The starting prices of the new sickle cell treatments are expected to range from $100,000 to over $1 million. It will be a challenge to make them accessible to the millions of people in poor countries who have sickle cell.

The disease is caused by a single variation in a gene for hemoglobin, the protein that carries oxygen in our red blood cells. The genetic trait is prevalent among those whose descent traces to sub-Saharan Africa, because a single copy of the sickle cell gene protects you from developing malaria. Inherit a copy from each parent, however, and you become one of 300,000 babies born in the world each year with sickle cell disease. It causes hemoglobin molecules to form long chains that warp red blood cells into sickle shapes that get stuck in blood vessels and block the flow to vital organs.

The result is terrible pain, organ damage, infections, andwhen left untreateddeath before age 5, on average. In the U.S., where the disease occurs in one of every 365 Black babies, available treatments still leave many with a life of pain, disability, and death before age 50.

The first sickle cell treatment approved by the U.S. Food and Drug Administration was hydroxyurea, in 1998. For the half of sickle cell patients with moderate disease, treatment with hydroxyurea, antibiotics, and transfusions can allow productive lives. Hydroxyurea is off-patent and costs less than a dollar a day. But even that price is beyond reach of those who live in resource-poor countries, says Russell Ware, a pediatric hematology professor at the University of Cincinnati College of Medicine. Ware is working with medical colleagues in Uganda to get more children there on hydroxyurea.

Data as of 7/22/20

Bloomberg; company filings

The beauty is that its off-patent, says Ware, but its also a curse, because no one can make money off it.

Novartis (NVS) is supporting hydroxyurea availability in collaboration with the government of Ghana. The Swiss drug giant is also planning clinical trials in Ghana and Kenya for Adakveo, a monoclonal antibody approved by the FDA last November as one of the first novel treatments in decades for sickle cell disease. Adakveo dampens the inflammatory process that makes sickle cells clog blood vessels in cases that send some 50,000 Americans to the emergency room every year.

The intravenous drugs initial sales in the years first half were $36 million, but analysts hope that by the middle of this decade, Adakveos annual sales could reach $1 billion to $2 billion in the U.S. and Europe.

Just days after approving the Novartis drug, the FDA approved another sickle cell drug called Oxbryta, from Global Blood Therapeutics (GBT). Oxbryta is a pill that prevents defective hemoglobin from forming chains within red blood cells. The product is GBTs first, and it produced $14 million in sales for the March quarter, with a loss of $73 million.

But analysts like Yatin Suneja of Guggenheim Securities say that Oxbryta can hit sales of $1.7 billion in a few years, yielding some $13 a share in earnings for GBT. That makes Suneja think that GBT stock can rise from its current level of $72 to $115.

Successful sickle cell treatments could lift a number of biotech companies.

GBT CEO Ted Love says that Oxbrytas benign safety profile gives the company confidence that it will win approval to market the product for children, and in higher doses for adults. If other sickle cell treatments in the companys pipeline pan out, he imagines that sickle cell might one day become as well managed a disease as HIV.

Love is aware of the potentially curative gene therapies being tested by others, but he thinks that patients with milder cases of sickle cell might say, Just give me a pill.

For several decades, it has been possible to cure sickle cell with a bone-marrow transplant from a related donor. But the scarcity of matched donors and the risk of serious immune reactions have limited the number of such procedures to about 1,000. Recent breakthroughs in genetic technology promise to overcome those limitations by extracting a patients own cells, manipulating the cells genetic code, and then replacing the patients marrow with the amended cells.

The Cambridge, Mass.based bluebird bio is already in Phase 3 clinical trials of its beti-cel gene therapy for another inherited disorder of red blood cells called beta-thalassemia. Less common than sickle cell, thalassemia leaves patients with so few red blood cells that they can need more than a dozen transfusions a year. At a recent online gathering of hematologists, bluebird said that 60 children and adults with thalassemia had gone through its beti-cel procedure. About 90% had gone a year without needing a single transfusion.

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With a similar treatment for sickle cell, which bluebird calls LentiGlobin, more than two dozen patients have been infused with their own modified cells. Among the 14 patients who were six months past their treatment, there has been a 99.5% decrease in the blood vessel jam-ups that the patients previously suffered.

That is fundamentally transformative, says bluebird CEO Nick Leschly, and far exceeds any and all expectations weve ever had, any of our investigators have ever had, or the patients that have been treated.

These potentially curative cell therapies are complex procedures that are expected to be priced at about $1 million a patient. The thalassemia treatment is already approved in Europe. After the patients in bluebirds U.S. trials for thalassemia and sickle cell have been followed up for 18 months, the company will seek FDA approvalhopefully next year.

RBC Capital Markets believes that the companys sickle cell treatment could reach sales of $2 billion a year and help lift bluebird stock from its recent price of $66 to $100.

It took bluebird a decade to get to this point, and its shares have sunk as low as $17 and soared as high as $236, as investors reacted to the companys dramatic successes against refractory cancers. With Buy recommendations all along Wall Street, bluebird will get the capital it needs to cross the finish line.

Hard on the heels of bluebird are companies that believe they have better genetic treatments. One of them is Crispr Therapeutics. The company takes its name from CRISPR, shorthand for a Nobel Prizewinning technology that homes in on a targeted stretch of DNA and snips the double-stranded molecular code with a kind of chemical scissors.

The way I describe it to my patients, says patient Grays doctor Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville, is that you have a book with 500 pages of thousands of words, and we are finding one word and correcting it.

Crispr Therapeutics CEO Sam Kulkarni says that CRISPR technology should provide more durable and uniform results than earlier generation treatments like bluebirds. When Crispr reported Grays healthy progress last month, it said that a second patient with sickle cell has also been treated, as well as five patients with thalassemia. Kulkarni hopes to reach the market with his treatments not too far behind bluebird.

This years surge in Crispr Therapeutics stock allowed it to raise about $450 million in a June stock offering. Jefferies analyst Maury Raycroft has estimated that a successful sickle cell treatment could contribute a third of the $5 billion in annual revenue he projects for Crispr by 2030, with earnings above $30 a share. The stock has blasted through his last price target of $82.

Behind Crispr are still other gene-editing companies working on treatments for thalassemia and sickle cell, including Sangamo Therapeutics (SGMO), Editas Medicine (EDIT), and Intellia Therapeutics (NTLA).

One of this years most successful initial public offerings was the February debut of Beam Therapeutics (BEAM), which is in preclinical testing of sickle and thalassemia treatments that would use a next-generation editing technology that CEO John Evans believes will be even more effective than CRISPR.

If these other companies sickle cell treatments pan out, they may be scrapping for market share after bluebird and Crispr have established products. Still, as the pharmaceutical giants shop for gene-editing know-how, there may be buyouts.

Sickle cell has been a long-neglected illness, but many companies are now competing with treatments that each hopes will be best-in-class. This should be a dogfight, says bluebird CEO Leschly, because thats in the interest of patients.

Write to Bill Alpert at

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These Companies Are Seeking a Cure for Sickle Cell. And Its Just the Beginning for Some Gene Therapy Stocks. - Barron's

FDA Approves Combination Therapy for Treating Advanced Melanoma – Pharmacy Times

Officials with the FDA have approved atezolizumab (Tecentriq, Genentech) plus cobimetinib (Cotellic, Genentech and Exelixis) and vemurafenib (Zelboraf, Genentech and Daiichi Sankyo) for the treatment of patients with BRAF V600 mutation-positive advanced melanoma. The safety profile observed in the atezolizumab combination was consistent with the known safety profiles of the individual medicines, according to Genentech.

Atezolizumab is a prescription monoclonal antibody designed to bind with a protein, PD-L1, expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. The therapy is currently utilized for treatment of patients with certain types of cancer, including urothelial carcinoma, non-small cell lung cancer, triple-negative breast cancer, and hepatocellular carcinoma.

The approval of the atezolizumab combination for BRAF V600 mutation-positive advanced melanoma is based on results from the Phase III IMspire150 study, a multi-center, double-blind, placebo-controlled randomized study in individuals with previously untreated BRAF V600 mutation-positive metastatic or unresectable locally advanced melanoma. The study results showed the addition of atezolizumab to cobimetinib and vemurafenib helped individuals live longer without their disease worsening or death (progression-free survival, PFS), compared to placebo plus cobimetinib and vemurafenib (median PFS 15.1 months versus 10.6 months respectively; hazard ratio, HR=0.78; confidence interval: 0.63-0.97; P=0.025).

The most common adverse reactions (rate 20%) in patients who received atezolizumab with cobimetinib and vemurafenib were rash (75%), musculoskeletal pain (62%), fatigue (51%), hepatotoxicity (50%), pyrexia (49%), nausea (30%), pruritus (26%), edema (26%), stomatitis (23%), hypothyroidism (22%), and photosensitivity reaction (21%).

The supplemental Biologics License Application or atezolizumab was granted under Priority Review. The review was also conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology products among international partners.

According to Genentech, the company has an extensive development program for atezolizumab, including multiple ongoing and planned phase 3 studies across lung, genitourinary, skin, breast, gastrointestinal, gynecological, and head and neck cancers. This includes studies evaluating atezolizumab both alone and in combination with other medicines.

For those who qualify, Genentech will offer patient assistance programs for patients prescribed atezolizumab with cobimetinib and vemurafenib by their doctor through Genentech Access Solutions.


FDA Approves Genentechs Tecentriq plus Cotellic and Zelboraf for People With Advanced Melanoma [news release]. South San Francisco, CA; July 30, 2020: Genentech website. Accessed July 30, 2020.

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FDA Approves Combination Therapy for Treating Advanced Melanoma - Pharmacy Times

How Can Technology Help Fight the COVID-19 Pandemic? – IoT For All

As the COVID-19 pandemic continues unfolding, technology solutions and government initiatives are multiplying to help monitor and control the viruss journey. Their aid includes reducing the load on the health system and reinforcing the efforts of overworking and burned-out healthcare workers.

While smart technologies cannot replace or compensate public institution measures, they do play a crucial role in emergency responses. Lets take a look at the promising use cases of how technology can help fight the novel coronavirus outbreak.

People tend to think of technology as a heartless machine, which is true, but only until its used for good. Just look at all the wonderful things weve managed to do with its help.

Telemedicine is gaining traction by offering remote patient monitoring and interactive remote doctors visits. At the same time, 3D printing and open-source solutions are facilitating the production of more affordable face masks, ventilators, and breathing filters as well as optimizing the supply of the medical equipment. Even more, the pandemic has driven scientists to desperate measures. They are now experimenting with gene editing, synthetic biology, and nanotechnology to develop and test vaccines faster than ever in the history of humanity.

Smart technologies like the Internet of things (IoT), big data, and artificial intelligence (AI) are being massively adopted to help track the disease spread and contagion, manage insurance payments, uphold medical supply chains, and enforce restrictive measures. Lets go step by step to see how IoT, AI, big data, and mobile solutions are actually enhancing medical care.

IoT has already found its use among healthcare providers. Today, connected patient imaging, health devices or applications, worker solutions, and ambulance programs are being adopted globally. But COVID-19 made the technology take on new applications to help the world combat the epidemic. Tracking quarantine, pre-screening and diagnosing, cleaning and disinfecting, innovative usage of drones, reducing in-home infections, are all new normals thanks to IoT.

For example, an American health technology companyKinsacreates smart thermometers that screen and aggregate peoples temperature and symptoms data in real-time. Having gathered data from over one million connected thermometers, Kinsa rolled out itsUS HealthWeather Map.

The map is updated daily, highlighting how severely the population is being affected by influenza-like illness (ILI). This real-time information helps health authorities see an increase. In fevers as early indicators of the community spread of COVID-19 to streamline the allocation of health resources. These areas are marked in the Atypical mode of the map.

To slow down the spread of COVID-19, a team of Seattle engineers createdImmutouch, a smart wristband vibrating every time a person wearing it tries to touch their face.

Smart speakers, lights, and security systems are being used to open doors and switch on lights to reduce in-home infections. These gadgets allow people to avoid touching the surfaces of doorknobs, switches, mail, packages, or anything that could easily spread germs.

Tapping into big data is a must to develop real-time forecasts and arm healthcare professionals with a profound database to help with decision-making.

IBMClinical Development system is an advanced Electronic Data Capture (EDC) platform that allows an accelerated delivery of medications to market and reduces the time and cost of clinical trials thanks to cognitive computing, patient data assets, and IoT. Additionally, the U.S. government had been in active talks with Facebook, Google, and others to determine how to use location data to glean insights for combating the COVID-19 pandemic.

The COVID-19 pandemic has become a game-changer for the healthcare continuum. Todays mobile apps are on guard to help patients receive online therapy, at-home testing, conclude self-checks, and improve mental well-being. Thanks to smartphone apps, it is now possible to trace the viruss journey and help limit its spread.

Apple COVID-19, for instance, was created in partnership with the Centers for Disease Control and Prevention (CDC), the White House, and the Federal Emergency Management Agency (FEMA). The application contains vital and relevant information from trusted sources on the coronavirus pandemic: hand hygiene practices, social distancing FAQs, quarantine guidelines, self-checking tutorials, tips on cleaning, and disinfecting surfaces. On top of that, it has a screening tool that advises people on what to do when a person has COVID-19 symptoms, has just returned from abroad, or has come in close contact with someone who might be infected with the disease.

Meanwhile, health authorities in Abu Dhabi have created theTraceCovidapp for Bluetooth-enabled smartphones to minimize the spread of the disease. The service allows tracing individuals who have come into proximity with a person tested positive for COVID-19. Thanks to it, medical professionals an react faster and render the necessary healthcare. Germany, in turn, is going to roll outa smartphone app, which will use Bluetooth to alert people if they are close to someone with the confirmed viral infection.

Telemedicine has also proved to be an efficient tool for flattening the curve.The Sheba Medical Centre, the largest Israeli hospital, launched a telehealth program for remote patient-monitoring to control the pandemic spread. Doctolib, a Franco-German company, Qare (France), Livi (Sweden), Push Doctor (the UK), Compugroup Medical (Germany) are offeringvirtual doctorstoo.

Artificial intelligence-powered by natural language processing (NLP) and location monitoring is crucial for identifying, tracking, and scanning outbreaks, predicting hotspots and helping make better decisions.

For example, Microsoft collaborated with the U.S. Centers for Disease Control and Prevention (CDC) to create an AI-basedCOVID-19 Assessment botto treat patients more effectively and allocate limited resources. The bot, nicknamed Clara, can evaluate symptoms, advice on the next steps to take and track users who need urgent care the most.

The Canadian startup BlueDot has applied AI to spot and track the spread of COVID-19 and predict outbreaks, and the Japanese company Bespoke rolled outBebot, an AI-powered chatbot that was developed specifically for travelers. This mobile app informs and assists them with coronavirus-related questions as they move about.

Theres no doubt that the coronavirus pandemic has become a real-life test for everyone. It has caused tremendous damage, but at the same time, it has forced tech innovators to roll out advanced solutions, and it seems that they dont plan on slowing down anytime soon.

Healthcare providers across the globe are continually switching to smart technologies. So if you are in the smart technology niche, consider the current trends to steer your business in the right direction.

How Can Technology Help Fight the COVID-19 Pandemic? - IoT For All

Thomas Jefferson University doctor to be honored at W&J commencement – Observer-Reporter

At its virtual commencement ceremony Saturday for the Class of 2020, Washington & Jefferson College will be presenting an honorary Doctor of Science degree to Mark L. Tykocinski, a medical doctor at Thomas Jefferson University in Philadelphia.

Tykocinski is the dean of the universitys Sidney Kimmel Medical College, one of the oldest in the nation. He has been in that role since 2008. Tykocinski is also the provost and executive vice president of academic affairs at the university. Before working at Thomas Jefferson University, Tykocinski spent a decade as a professor and chair of the Department of Pathology and Laboratory Medicine at the University of Pennsylvania. His research interests have been in the fields of cellular and molecular immunology, cell surface engineering and biologics innovation.

Tykocinski has contributed to the development of novel cell therapy strategies and the engineering of a novel class of mammalian gene expression vectors that have been distributed around the world. He holds research patents in the fields of molecular and cellular immunology, and is a fellow with the National Academy of Inventors. He received degrees from Yale and New York universities.

The college that Tykocinski oversees is closely linked to W&J. The Class of 2020 at W&J includes the first students to receive degrees after completing a year of study at the medical school. This is happening thanks to a partnership W&J struck up with the Sidney Kimmel Medical College that allows W&J students with majors outside the natural sciences to start studying at Sidney Kimmel after their junior year at W&J. Thomas Jefferson University started the partnership as part of the universitys efforts to bring more liberal arts students into their medical college.

W&J had hoped to hold an in-person commencement ceremony Saturday, but changed it to an online event due to rising COVID-19 numbers in the region. The college is hoping to hold an in-person reunion for members of the Class of 2020 at some point, perhaps as soon as next year.

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Thomas Jefferson University doctor to be honored at W&J commencement - Observer-Reporter

First Edition: July 27, 2020 – Kaiser Health News

Today's early morning highlights from the major news organizations.

Kaiser Health News:Its About Love And Solidarity: Mutual Aid Unites NYC Neighbors Facing COVIDNancy Perez, a 45-year-old resident of the Brooklyn neighborhood of Bedford-Stuyvesant, contracted COVID-19 in March. She stayed quarantined in her room for a month to isolate from her two sons and grandson. A few days before she got the virus, shed met a volunteer with Bed-Stuy Strong one of the many mutual aid groups around the country that have rallied to provide help in the face of the pandemic. Bed-Stuy Strong assembled an army of volunteers to help vulnerable neighbors with food deliveries and basic supplies. While Perez was in isolation, volunteers regularly delivered cooked food for her sons, ages 17 and 20, and her 4-year-old grandson. (Lawrence, 7/27)

Kaiser Health News:The Color Of COVID: Will Vaccine Trials Reflect Americas Diversity?When U.S. scientists launch the first large-scale clinical trials for COVID-19 vaccines this summer, Antonio Cisneros wants to make sure people like him are included. Cisneros, who is 34 and Hispanic, is part of the first wave of an expected 1.5 million volunteers willing to get the shots to help determine whether leading vaccine candidates can thwart the virus that sparked a deadly pandemic. If I am asked to participate, I will, said Cisneros, a Los Angeles cinematographer who has signed up for two large vaccine trial registries. It seems part of our duty. (JoNel Aleccia, 7/27)

Kaiser Health News:Employers Require COVID Liability Waivers As Conflict Mounts Over Workplace SafetyAfter spending a May day preparing her classroom to reopen for preschoolers, Ana Aguilar was informed that the tots would not have to wear face masks when they came back. Whats more, she had to sign a form agreeing not to sue the school if she caught COVID-19 or suffered any injury from it while working there. Other teachers signed the form distributed by the Montessori Schools of Irvine, but Aguilar said she felt uncomfortable, although it stipulated that staff members would be masked. At 23, she has a compromised immune system and was also worried that she could pass the coronavirus on to her fianc and other family members. (Meyer, 7/27)

Kaiser Health News:Last Thing Patients Need During Pandemic: Being Last To Know A Doctor Left NetworkAs the coronavirus spread silently through New York City early this year, Deborah Koeppel had an appointment with her cardiologist and two visits with her primary care doctor. Both physicians are members of Concorde Medical Group, a practice in Manhattan with an office conveniently located a few blocks from where Koeppel works. She soon received notices telling her after the fact that those doctors were not in her health plans network of providers. According to the notices, she was on the hook for $849 in out-of-network cost sharing for three visits, which typically would cost her nothing from in-network providers. (Andrews, 7/27)

NPR:Florida Case Count Surpasses That Of New York, The Country's Original EpicenterFlorida has recorded more coronavirus cases than New York. Only California, the most populous state in the country, has more. As of Sunday afternoon, data from Johns Hopkins University shows 423,855 people in Florida have tested positive for the coronavirus, compared to 411,736 in New York. California leads with 450,242 cases. (Treisman, 7/26)

Tampa Bay Times:Florida Coronavirus Cases Show Little Sign Of Slowing As State Surpasses New YorkThe number of infections and deaths tied to the novel coronavirus in Florida showed little sign of slowing Sunday as the state surpassed New York for the second-highest number of confirmed cases in the United States. Only California, with a population nearly twice as high as Floridas, has more cases. Floridas Department of Health reported 9,344 infections and 78 fatalities. The overall caseload is 423,855 since March 1, and the number of deaths tied to the virus is 5,972. (Dawson, 7/26)

The Hill:US Surpasses 1,000 COVID-19 Deaths For Fourth Straight DayThe U.S. tallied over 1,000 coronavirus-related deaths Friday for the fourth straight day this week, yet another sign of the alarming spike in COVID-19 cases across the country.There were 1,178 new deaths Friday alone, according to the COVID Tracking project, compared with 1,038 Tuesday, 1,117 Wednesday, and 1,039 Thursday. Over 137,000 people have died in the U.S. and over 4 million people have contracted the virus in the country since the outbreak began. (Axelrod, 7/25)

NPR:U.S. Coronavirus Hot Spots: Mid-Atlantic And Northeast Could BackslideFor weeks the U.S. coronavirus pandemic has largely been driven by spiraling outbreaks in the South and West. But some forecasters say Mid-Atlantic and Northeast states could soon be in deep trouble again, too. The warning comes from researchers at the PolicyLab at Children's Hospital of Philadelphia, which has built a model to provide four-week forecasts for every U.S. county. NPR spoke to David Rubin, PolicyLab's director, an epidemiologist and professor at the University of Pennsylvania. (Aizenman, 7/24)

Stat:Trump Unveils Four Executive Orders Aimed At Lowering Drug PricesThe Trump administration unveiled four executive orders on Friday aimed at bringing down pharmaceutical prices, a last-ditch effort by the White House to cut drug costs before the November election. It remains unclear whether the Trump administration is capable of finalizing many of the actions by Election Day and whether it intends to do so. (Florko and Facher, 7/24)

Politico:Trump Signs Limited Drug Pricing Orders After Last-Minute Debate"The four orders I'm signing today will completely restructure the prescription drug market," Trump said in a speech, hearkening back to his 2016 campaign promise to slash costs. But the ambitious plans are rife with limitations. The rebate order comes with a caveat that any plan cannot increase seniors' premiums, the unworkable problem that led the adminstration to kill its original rebate rule last year. (Owermohle, Cancryn and Luthi, 7/24)

Politico:Trumps Talking Health Care Again, With 2020 In MindPresident Donald Trump is suddenly talking about health care again. He signed several executive orders on drug pricing on Friday. He vowed to unveil some new health plan by the end of next week, although he hasnt provided specifics or an explanation of how hell do it. His aides are touting a speech in which Trump will lay out his health care vision. White House counselor to the president Kellyanne Conway has been calling Trump the health care president. (McGraw and Ehley, 7/26)

AP:AP-NORC Poll: US Course At Record Low, Trump Sinks On VirusA new poll from The Associated Press-NORC Center for Public Affairs Research also finds Trumps approval for his handling of the COVID-19 pandemic falling to a new low, with just 32% of Americans supportive of his approach. ... Even as he tries to refocus his contest with Biden on divisive cultural issues and an ominous law and order message, Trumps reelection prospects are likely to be inextricably linked to his handling of the pandemic and whether voters believe the country will head back in the right direction under his leadership. The AP-NORC poll makes clear the challenge ahead for Trump on that front: 8 in 10 Americans say the country is heading in the wrong direction. (Pace and Fingerhut, 7/26)

Politico:Make America Normal Again: Trump Backers Plead For A Virus PlanPresident Donald Trump restarted the White House coronavirus briefings. He urged Americans to wear masks. He even scrapped his partys convention. To many of his own allies, its still not enough. Trumps political allies, alarmed by his sinking poll numbers, are warning that the presidents best chance to get reelected is to outline more detailed plans to conquer the coronavirus he keeps trying to wish away. They are advising him to offer people something concrete they can look to as the pandemic surges in dozens of states, eroding months of progress. (Kumar, 7/24)

The Wall Street Journal:GOP To Propose Aid Bill, With Extra Jobless Benefits Set To ExpireAfter days of disagreements between the White House and GOP lawmakers, Republicans are set to release their proposal for the next coronavirus relief bill on Monday, with millions of Americans on the verge of losing expanded unemployment benefits.Lawmakers now have little time before the $600 weekly supplement to jobless benefits ends. In negotiations with Democrats, three months before the election, an agreement on unemployment insurance might prove to be the most difficult to reach. (Ballhaus and Duehren, 7/26)

The Hill:White House, Senate GOP Race To Finalize Coronavirus Package Ahead Of Monday RolloutThe White House and Senate Republicans are workingto finalize a coronavirus relief package ahead of a Monday rollout. Treasury Secretary Steven Mnuchin and White House chief of staff Mark Meadows were back in the Capitol on Sunday for a second day of meetings with GOP staff as they work to lock down the forthcoming proposal. As he left the Capitol, Meadows told reporters that they had"been working through just some of the technical language" and had reached "an agreement in principle." (Carney, 7/26)

The Hill:Mnuchin: It 'Wouldn't Be Fair To Use Taxpayer Dollars To Pay More People To Sit Home'Treasury Secretary Steven Mnuchin took a hard line Sunday against the $600 increase in unemployment benefits that was a part of the last coronavirus relief measure, saying, It just wouldnt be fair to use taxpayer dollars to pay more people to sit home than they would working and get a job. GOP lawmakers have taken a hard line against theenhancement as they negotiate with the White House over a new relief measure. The initial bill won blowback from Republicans who said some people would make more money not working than going to work. (Budryk, 7/26)

The New York Times:Trump Officials Float Idea Of Narrow Bill To Extend Unemployment BenefitsTop Trump administration officials proposed on Sunday potentially short circuiting free-ranging stimulus talks with Democrats to rush through a much narrower bill prioritizing an extension of federal unemployment benefits that are set to expire this week for millions of Americans. Mark Meadows, the White House chief of staff, said he would now like to see lawmakers act this week to extend and alter the unemployment program, give tax credits to businesses to help ease reopening costs and grant employers new liability protections while setting aside a long list of other objectives, including Democrats priorities. (Fandos and Cochrane, 7/26)

AP:White House Pushes Narrow Virus Aid; Pelosi Blasts GOP DelayHouse Speaker Nancy Pelosi on Sunday assailed Republican disarray over a new pandemic relief package as the White House suggested a narrower effort might be necessary, at least for now. The California Democrat panned the Trump administrations desire to trim an expiring temporary federal unemployment benefit from $600 weekly to about 70% of pre-pandemic wages. The reason we had $600 was its simplicity, she said from the Capitol. (Mascaro and Superville, 7/26)

The New York Times:Fires And Pepper Spray In Seattle As Police Protests Widen Across U.S.Weeks of violent clashes between federal agents and protesters in Portland, Ore., galvanized thousands of people to march through the streets of American cities on Saturday, injecting new life into protests that had largely waned in recent weeks. One of the most intense protests was in Seattle, where a day of demonstrations focused on police violence left a trail of broken windows and people flushing pepper spray from their eyes. At least 45 protesters had been arrested as of early evening, and both protesters and police officers suffered injuries. (Baker and Bogel-Burroughs, 7/25)

AP:Police And Protesters Clash In Violent Weekend Across The USProtests took a violent turn in several U.S. cities over the weekend with demonstrators squaring off against federal agents outside a courthouse in Portland, Oregon, forcing police in Seattle to retreat into a station house and setting fire to vehicles in California and Virginia. A protest against police violence in Austin, Texas, turned deadly when a witness says the driver of a car that drove through a crowd of marchers opened fire on an armed demonstrator who approached the vehicle. And someone was shot and wounded in Aurora, Colorado, after a car drove through a protest there, authorities said. (Martin, 7/26)

Reuters:Seattle Black Lives Matter Clashes Spark 45 Arrests, 21 Police InjuredDozens were arrested and many police injured in clashes around Seattles biggest Black Lives Matter protest in weeks on Saturday, police said, with a renewed energy sparked by violent clashes between activists and federal agents in nearby Portland, Oregon. Police said officers used non-lethal weapons in attempts to disperse the thousands of marchers in the late afternoon after some protesters set fire to the construction site for a King County juvenile detention facility and courthouse. By 10 p.m., police had made 45 arrests in connection with todays riot in the East Precinct, the Seattle Police wrote in a Twitter post. Scruggs, 7/25)

The New York Times:Hurricanes Choice For Texans: Shelter From The Virus Or The StormBartt Howes boat was his refuge from the pandemic. Battling diabetes and H.I.V., he knew that catching the coronavirus as well could kill him, so he had been living alone on the docked boat for three months. Then Hurricane Hanna began to slam the Texas coast on Saturday, forcing Mr. Howe to trade one deadly menace for another: To avoid injury or death in the hurricane, he had to risk infection ashore. I had managed to stay safe all this time, but the storm kicked me out of my boat, he said with a hint of resignation. Now here I am, back on land, on borrowed time. (Sandoval, 7/26)

AP:Tropical Storm Hanna Drenches South Texas Amid Virus CrisisA day after roaring ashore as a hurricane, Hanna lashed the Texas Gulf Coast on Sunday with high winds and drenching rains that destroyed boats, flooded streets and knocked out power across a region already reeling from a surge in coronavirus cases. Downgraded to a tropical storm, Hanna passed over the U.S.-Mexico border with winds near 50 mph (85 kph), the National Hurricane Center said. It unloaded more than 12 inches (30 centimeters) of rain on parts of South Texas and northeastern Mexico. Border communities whose health care systems were already strained by COVID-19 cases with some patients being airlifted to larger cities found themselves under siege from the first hurricane of the 2020 Atlantic season. There were no immediate reports of any deaths on either side of the border. (Mone and Merchant, 7/26)

The Washington Post:Tropical Storm Hanna Unloading Flooding Rains In South TexasOn Sunday evening, Hanna continued its march southwest as a tropical depression, slipping into Mexico while still lashing the Rio Grande Valley with prolific rainfall. The direct strike by Hanna comes at a time when the Lone Star State is grappling with a spike in coronavirus cases. (Cappucci, Samenow and Freedman, 7/26)

The New York Times:FEMA Sends Faulty Protective Gear To Nursing Homes Battling VirusExpired surgical masks. Isolation gowns that resemble oversize trash bags. Extra-small gloves that are all but useless for the typical health workers hands. Nursing home employees across the country have been dismayed by what theyve found when theyve opened boxes of protective medical gear sent by the federal government, part of a $134 million effort to provide facilities a 14-day supply of equipment considered critical for shielding their vulnerable residents from the coronavirus. (Jacobs, 7/24)

Politico:Trump Administration Invests $472M More In Moderna Vaccine CandidateThe Trump administration is going to pump another $472 million into expanding Modernas clinical trial to test the safety and efficacy of its coronavirus vaccine candidate. What happened: Moderna announced Sunday that the Biomedical Advanced Research and Development Authority, known as BARDA, is pouring the additional dollars the day before the phase three trial of the vaccine candidate is slated to start. (Roubein, 7/26)

Reuters:Moderna Gets Further $472 Million U.S. Award For Coronavirus Vaccine DevelopmentThe U.S.-based drug maker said the additional funding will support its late-stage clinical development including the expanded Phase 3 study of Modernas vaccine candidate. In April, Moderna had received $483 million from the U.S. federal agency that funds disease-fighting technology, when the experimental vaccine was in an early-stage trial conducted by the U.S. National Institutes of Health. (7/26)

The Hill:Meadows Says White House Is 'Hopeful' It Can Announce New Coronavirus Therapies 'In The Coming Days'White House chief of staff Mark Meadows said Sunday that the administration is hopeful it can announce new therapies to treat the coronavirus in the coming days. Meadows told ABCs This Week that the White House has been working around the clock,with a focuson COVID-19 therapeutics, vaccines and mitigation therapies.The president has been very clear whatever amount of money and whatever amount of time needs to be invested, were doing that, the White House chief of staff said. (Coleman, 7/26)

The Hill:Azar: If We Wear Masks, We Can Avoid Further ShutdownsHealth and Human Services Secretary Alex Azar said Sunday that widespread social distancing and mask usage would eliminate the need for resuming shutdowns due to the coronavirus pandemic. If we wear our masks we can avoid further shutdowns but if we dont that will be the consequence, Azar said Sunday on CBS Face the Nation. (Budryk, 7/26)

Politico:Azar Blames Testing Delays On StatesHHS Secretary Alex Azar Sunday blamed the current delays in coronavirus testing on the states, which he said have been too slow to spend federal dollars to boost the countrys testing amid the virus's spread. The Trump administration has frequently sought to put the responsibility for the coronavirus response on governors and local officials, even as many public health officials as well as governors have called for a coordinated national emergency response. (Roubein, 7/26)

Politico:U.S. Testing Czar: Everyone Who 'Needs' A Covid-19 Test Can Get OneAdmiral Brett Giroir, the Trump administration coronavirus testing czar, said that anyone who needs a coronavirus test can get one but he acknowledged that the average turnaround time for tests is too long as states smash records for numbers of cases. Appearing on CNN's "State of the Union," he pushed back at former Trump chief of staff Mick Mulvaney who earlier this month called his familys difficulties obtaining tests promptly inexcusable" this many months into the pandemic. (Roubein, 7/26)

The Hill:Ex-CDC Director On US, COVID-19: 'We Are A Laggard'Former Centers for Disease Control and Prevention Director Tom Frieden on Sunday said the U.S. had been a laggard in addressing the coronavirus pandemic, specifically pointing to lack of centralized information. Ill be frank, we are a laggard, Frieden said on Fox News Sunday. We are one of the top in the world in terms of the cumulative death rate unlike many other countries that have high death rates, ours is continuing to increase. (Budryk, 7/26)

The Hill:CDC: Even Mild Coronavirus Symptoms Can Persist For WeeksCOVID-19 can result in prolonged illness even in people with mild symptoms, including young adults, according to a newanalysisreleased Friday by the Centers for Disease Control and Prevention (CDC).It has been known that people severely ill after contracting COVID-19 can stay sick for several weeks. But less has been known about theeffects of the disease on people with milder symptoms who dont require hospitalization. (Hellmann, 7/24)

Stat:Covid-19 Vaccines May Cause Mild Side Effects, Experts SayWhile the world awaits the results of large clinical trials of Covid-19 vaccines, experts say the data so far suggest one important possibility: The vaccines may carry a bit of a kick. In vaccine parlance, they appear to be reactogenic, meaning they have induced short-term discomfort in a percentage of the people who have received them in clinical trials. This kind of discomfort includes headache, sore arms, fatigue, chills, and fever. As long as the side effects of eventual Covid-19 vaccines are transient and not severe, these would not be sources of alarm in fact, they may be signals of an immune system lurching into gear. (Branswell, 7/27)

The New York Times:Your Coronavirus Antibodies Are Disappearing. Should You Care?Your blood carries the memory of every pathogen youve ever encountered. If youve been infected with the coronavirus, your body most likely remembers that, too. Antibodies are the legacy of that encounter. Why, then, have so many people stricken by the virus discovered that they dont seem to have antibodies? Blame the tests. (Mandavilli, 7/26)

The Wall Street Journal:A Big Unknown In Covid-19 Vaccine Development: How Long Will Protection Last?If any of the most-advanced Covid-19 vaccines prove to work safely, they may protect people for months or years rather than the rest of their lives, according to emerging science and health experts. Only a handful of vaccines generate lifetime immunity for most people, such as the ones for measles, a viral infection that naturally produces lifelong immunity. Experts caution against expectations of such longevity for Covid-19, citing experience with other respiratory viruses plus emerging data on the longevity of the antibodies that can prevent the virus from entering human cells and replicating. (Hopkins, Hernandez and Loftus, 7/26)

The Washington Post:Coronavirus Vaccine Trials Aim To Include The Black And Hispanic CommunitiesEach fall, the Rev. Rob Newells urges the congregation at Imani Community Church in Oakland, Calif., to get a flu shot. He builds bridges everyday between the countrys most vulnerable, marginalized communities and the medical system, defusing suspicion about HIV prevention treatments and educating people about medical research. He prods health-care leaders to think harder about their messengers: Dont send a white doctor to tell black people what they need to do for their own good. But with the first massive coronavirus vaccine trial in people set to start Monday, Newells finds himself in an unfamiliar place: on the fence about what to tell his colleagues, his community, his cousins. Biomedical research, Newells knows, is a long and painstaking process and he is concerned about a vaccine campaign that seems so narrowly focused on speed. (Johnson, 7/26)

Stat:Covid-19 Surge Helps AI Researchers Amass Lung ScansAt first, the images of lungs infected by the novel coronavirus were hard to come by. It was early in the pandemic, and Joseph Paul Cohen, a researcher at the University of Montreal, was trying to stockpile radiology scans to train an artificial intelligence model to recognize warning signs of severe illness. With so few images available, the work was next to impossible. But in recent weeks, the resurgence of Covid-19 in the U.S. and other hotspots has solved that problem, allowing him to amass hundreds of lung scans from clinical reports published around the world. (Ross, 7/27)

The New York Times:Corporate Insiders Pocket $1 Billion In Rush For Coronavirus VaccineOn June 26, a small South San Francisco company called Vaxart made a surprise announcement: A coronavirus vaccine it was working on had been selected by the U.S. government to be part of Operation Warp Speed, the flagship federal initiative to quickly develop drugs to combat Covid-19. Vaxarts shares soared. Company insiders, who weeks earlier had received stock options worth a few million dollars, saw the value of those awards increase sixfold. And a hedge fund that partly controlled the company walked away with more than $200 million in instant profits. (Gelles and Drucker, 7/25)

The Washington Post:ADHD Video Game Treatment Approved By FDACan a video game help children struggling with ADHD? That question inspired hopeful headlines last month after the Food and Drug Administration permitted marketing of the first digital game that may be prescribed to treat children ages 8 to 12 who have been diagnosed with attention-deficit/hyperactivity disorder. In EndeavorRx, designed for iPhones and iPads, children guide an avatar surfing through molten lava and an icy river, dodging fires and icebergs while grabbing flying objects. The game is not yet available for purchase, nor has a price been released, but its Boston-based developer, Akili Interactive Labs, may now feature its unique status in ads and pursue coverage by insurance plans. (Ellison, 7/26)

CIDRAP:Study Finds No Transmission Of COVID-19 From Moms To NewbornsA study yesterday in The Lancet Child & Adolescent Health found no evidence of COVID-19 transmission between 120 babies born to mothers with COVID-19, even after 2 weeks of breastfeeding with appropriate hygiene precautions. The findings led the American Academy of Pediatrics (AAP) to change its guidance on infants and COVID-19positive mothers. The guidelines now recommend that newborns "room-in" with infected others after delivery if proper hygiene precautions are taken, including wearing a mask when appropriate and practicing hand hygiene. (7/24)

CIDRAP:Pilot Study Evaluates Use Of Dogs For SARS-CoV-2 DetectionA small pilot study suggests trained scent-detection dogs have the potential to be used for mass detection of people infected with SARS-CoV-2, the virus that causes COVID-19, German researchers reported yesterday in BMC Infectious Diseases. In the study, eight dogs were trained for 1 week to detect SARS-CoV-2 from saliva or tracheobronchial secretions of patients infected with the virus. During the training, dogs were presented with positive and negative samples (confirmed by RT-PCR tests) using a device with seven scent holes with tubes leading to metal containers that held the samples. Only one hole had a container with a positive sample, and the other six had containers with control samples. After a week, the researchers conducted a double-blind, randomized controlled trial. (7/24)

AP:US Agency Vows Steps To Address COVID-19 InequalitiesIf Black, Hispanic and Native Americans are hospitalized and killed by the coronavirus at far higher rates than others, shouldnt the government count them as high risk for serious illness? That seemingly simple question has been mulled by federal health officials for months. And so far the answer is no. But federal public health officials have released a new strategy that vows to improve data collection and take steps to address stark inequalities in how the disease is affecting Americans. Officials at the Centers for Disease Control and Prevention stress that the disproportionately high impact on certain minority groups is not driven by genetics. (Stobbe, 7/25)

USA Today:Diabetes And COVID: Coronavirus Highlights America's Health ProblemsDr. Anne Peters splits her mostly virtual work-week between a diabetes clinic on the west side of Los Angeles and one on the east side of the sprawling city.Three days a week she treats people whose diabetes is well controlled. They have insurance, so they can afford the newest medications and blood monitoring devices. They can exercise and eat well. Those generally more affluent West LA patients who've gotten COVID-19 have developed mild to moderate symptoms feeling miserable, she said but treatable, with close follow-up at home. ... On the other two days of her work week, it's a different story. (Weintraub, 7/27)

The New York Times:In Era Of Sickness, Doctors Prescribe Unusual Cure: VotingThe sign is easy to miss in the waiting room of the emergency department at Massachusetts General Hospital, next to the reception desk and a hand sanitizer pump. Register to vote here, it says, above an iPad attached to a podium. The kiosk has stood there since November, before the pandemic began and stayed there through the worst weeks of April, when 12 gasping patients were put on ventilators during a single grueling 12-hour shift. Now, as the number of coronavirus patients has slowed to a trickle, Dr. Alister Martin, the 31-year-old emergency room doctor who built the kiosk, is determined to keep trying to register voters. (Stockman, 7/25)

NPR:Gene Therapy Sees Encouraging Success In Child With Duchenne Muscular DystrophyThis is the story of a fatal genetic disease, a tenacious scientist and a family that never lost hope. Conner Curran was 4 years old when he was diagnosed with Duchenne Muscular Dystrophy, a genetic disease that causes muscles to waste away. Conner's mother, Jessica Curran, remembers some advice she got from the doctor who made that 2015 diagnosis: "Take your son home, love him, take him on trips while he's walking, give him a good life and enjoy him because there are really not many options right now." (Hamilton, 7/27)

NPR:Could A Flu Shot Reduce Your Alzheimer's Risk?For years, public health officials have been trying to dispel the myth that people who get a flu shot are more likely to get Alzheimer's disease. They are not. And now there is evidence that vaccines that protect against the flu and pneumonia may actually protect people from Alzheimer's, too. The evidence comes from two studies presented Monday at this year's Alzheimer's Association International Conference, which is being held as a virtual event. (Hamilton, 7/27)

The Washington Post:References To White Men Still Dominate College Biology Textbooks, Survey SaysCharles Darwin. Carolus Linnaeus. Gregor Mendel. Theyre all men. Theyre all white. And their names appear in every biology book included in a new analysis of college textbooks. According to the survey, mentions of white men still dominate biology textbooks despite growing recognition in other media of the scientific contributions of women and people of color. The good news, the researchers say: Scientists in textbooks are getting more diverse. The bad news: If diversification continues at its current pace, it will take another 500 years for mentions of black/African American scientists to accurately reflect the number of black college biology students. (Brookshire, 7/26)

NPR:ADA At 30: 'We Are Not The Ones That Need To Change'Before the Americans with Disabilities Act granted people with disabilities greater protection and accessibility, a little-known law set the groundwork. In 1977, Judy Heumann helped lead a peaceful protest that forced the government to follow through with Section 504. As part of the 1973 Rehabilitation Act, the law would force hospitals, universities and other public spaces that received federal money, to remove barriers to accessibility for all Americans. But its implementation was long delayed over the costs necessary to retrofit buildings to comply with the law. (Shapiro and Bowman, 7/26)

AP:TV Reporter Credits Viewer With Noticing Cancerous LumpA television news reporter in Florida is crediting an eagle-eyed viewer for noticing a lump on her neck and emailing her that she should get it checked out. Victoria Price, a reporter for WFLA in Tampa, followed the advice and was diagnosed with cancer. Price tweeted that she is undergoing surgery on Monday to remove the tumor, her thyroid and a couple of lymph nodes. (7/26)

Politico:Sinclair To Delay Segment Featuring 'Plandemic' Conspiracy TheorySinclair Broadcasting on Saturday said it will delay its scheduled airing of a news segment featuring a viral conspiracy theory surrounding Anthony Faucis role in the Covid-19 pandemic.America This Week host Eric Bolling was scheduled to air an interview with Judy Mikovits, a medical researcher featured in the Plandemic video that claims Fauci, the nations top infectious disease expert, was responsible for the creation of the coronavirus, Media Matters reported. (Eliza Weaver, 7/25)

The Wall Street Journal:Sinclair Postpones Controversial Show About CoronavirusSinclair Broadcast Group Inc., the owner of 191 television stations across the U.S., delayed the airing of an interview about the alleged origins of the coronavirus pandemic that drew widespread criticism on social media. An episode of America This Week, which was slated to air over the weekend, features an interview with medical researcher Judy Mikovits, who has claimed that Dr. Anthony Fauci, the governments top infectious-disease expert, helped manufacture the coronavirus and spread it to China. Dr. Mikovitss claims have previously attracted attention in the documentary Plandemic, which was earlier pulled from major online platforms including Facebook Inc. and Alphabet Inc.s YouTube. (Rizzo, 7/26)

AP:Workers Praise Disney Virus Safety, But Will Visitors Come?Every week, it seems, Kaila Barker, her husband and their five children change their minds about whether to travel from their home in Connecticut to Floridas Walt Disney World as planned in September. On the one hand, the lack of crowds means more opportunities to go on rides without long waits. On the other hand, Connecticut and Florida have implemented pandemic-related quarantines for each others residents and visitors, and the Barkers worry whether the Disney magic will get lost with mandatory mask-wearing for visitors and workers, temperature checks and no parades, fireworks shows or up-close meet-and-greets with costumed characters. (Schneider, 7/26)

The Wall Street Journal:Major Truck-Stop Chains Will Require Drivers To Use Face MasksThe biggest U.S. truck-stop operators will require customers to wear masks starting next week, joining major retailers, restaurants and airlines in rolling out policies aimed at reducing the spread of coronavirus. Pilot Co., which operates 780 travel centers under the Pilot Flying J and other brands, said its mandate will take effect July 28. Similar policies kick in July 29 at Loves Travel Stops & Country Stores Inc. and TravelCenters of America Inc. sites. (Smith, 7/24)

USA Today:Walmart Mask Incident In Minnesota: Pair With Swastikas Banned A YearA Minnesota man and woman who wore face masks with swastikas on them in an incident captured on video have been banned fromWalmart storesnationwide for at least a year.The video, posted to Facebook on Saturday byRaphaela Mueller, shows a man and woman in a Walmart in Marshall, Minnesota, wearing red face coverings with swastikas. The woman flips off the camera while the man checks out groceries. (Culver, 7/26)

AP:Some US Police Resist Enforcing Coronavirus Mask MandatesLang Holland, the chief of police in tiny Marshall, Arkansas, said he thinks the threat of the coronavirus has been overstated and only wears a face mask if hes inside a business that requires them. He doesnt make his officers wear them either. So the day after Republican Gov. Asa Hutchinson signed an order requiring masks to be worn in public throughout Arkansas, Holland made it clear his department wasnt going to enforce the mandate in the Ozarks town of about 1,300, calling it an unconstitutional overreach. (DeMillo, 7/26)

AP:Amid Virus, Uncertainty, Parents Decide How To School KidsJoshua Claybourn is leaning toward sending his kindergarten daughter to in-person classes at a private school next month. Holly Davis sixth-grade daughter will learn online, though the family has not yet decided what to do for school for a teenage daughter who requires special accommodations for hearing problems and dyslexia and another whos starting college. As they decide how their children will learn this fall amid the coronavirus pandemic, parents are anxiously weighing the benefits of in-person instruction against the risks that schools could shut their doors again or that their children could contract the virus and pass it on. (Webber and Groves, 7/26)

The Hill:McEnany Likens Schools To 'Essential Places Of Business' In Push For ReopeningThe White House would support sending children back to school even if future studies showed kids transmitCOVID-19 at a higher rate than currently known, press secretary Kayleigh McEnany said Friday, arguing schools are "essential places of business." McEnany fielded multiple questions from reporters about President Trump's push for a return to in-person learning this fall even as he cancels some events for the Republican National Convention due to concerns about holding a mass gathering during the pandemic. Deborah Birx, the White House coronavirus response coordinator, also said Friday it's "an open question" how rapidly children under the age of 10 spread the virus. (Samuels, 7/24)

AP:Colleges Plan For Virus Testing, But Strategies Vary WidelyFor students heading to Colby College in Maine this fall, coronavirus testing is expected to be a routine part of campus life. All students will be required to provide a nasal swab every other day for two weeks, and then twice a week after that. All told, the college says it will provide 85,000 tests, nearly as many as the entire state of Maine has since the pandemic started. Colby, a private school of 2,000 students, joins a growing number of colleges announcing aggressive testing plans to catch and isolate COVID-19 cases before they spread. Harvard University says all students living on campus will be tested when they arrive and then three times a week. Boston University plans to test most students at least once a week. (Binkley, 7/26)

The New York Times:Tailors Know New Yorkers Pandemic Secret: Everybody Got Fat!With weddings postponed and offices shut, business was bleak at Woodside Tailor Shop in Queens during the long months of pandemic lockdown. There was no need for party dress alterations, or any pressure for slacks to be hemmed. But about three months in, things started picking back up in June, with one particular service in sudden demand: People needed a bit more breathing room in their clothing. Everybody got fat! said Porfirio Arias, 66, a tailor at Woodside. Its not only in New York. Its all over the world that people got fat. (Maslin Nir, 7/25)

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First Edition: July 27, 2020 - Kaiser Health News

These are the most valued tech startups in France in 2020 – Silicon Canals

The tech startup ecosystem in France is witnessing a spectacular growth, thanks to the fresh pool of aspirational entrepreneurs and investors. This substantial growth has led to numerous fundraising rounds in the country that take these French tech startups to the next level even during the pandemic crisis. Over the past few months, even while France was one of the worst-hit markets due to the COVID-19 outbreak, the investments in tech startups did not cease.

As per a report by Pitchbook, the most valued French tech startups have a joint value of over 6 billion. These companies operate across various industries including Artificial Intelligence, insurtech, and healthcare. Without further ado, heres the list of most valued tech startups in France so far.

Founder/s: Francis Nappez, Nicolas Brusson, Frederic MazzellaFounded year: 2006Funding: 405 million

Paris-based BlaBlaCar is a long-distance ride-sharing network. The carpooling service lets users connect with drivers who have empty seats and book seats online. As it allows commuters to share the ride, the cost per person is relatively lower than travelling alone.

Recently, the French carpooling platform that recently joined hands with Voi Technology to help make scooters reliable and easily available across big cities in France. As this move was during the COVID-19 crisis, the company had to launch the BlaBlaRide service adhering to the local regulations and guidelines.

Back in November 2018, BlaBlaCar secured nearly 100 million later stage VC funding from Socit Nationale des Chemins de fer Franais, Omnes Capital and Insight Partners, thereby reaching a valuation of 1.43 billion.

Founder/s: Jessy Bernal, Ivan Schneider, Stanislas Niox-ChateauFounded year: 2013Funding: 237 million

Doctolib is a French healthtech startup, which offers a software solution to doctors and hospitals. It is basically a doctor appointment booking app, which comes with an entire range of services that help refine and improve the efficiency of medical operations.

The subscription-based online medical appointment management service attained the unicorn status early in 2019 following the investment from General Atlantic, Bpifrance, Accel and others. Well, Doctolib secured 150 million funding, which increased its valuation to 1.13 billion.

Founders: Guillaume Lestrade, Thomas RebaudFounded year: 2016Funding: 269 million

French AI startup Meero aims to simplify the work of the photographer. From providing revenues and market research to invoicing, post-production, and delivery, Meero takes care of all the complicated, time-consuming tasks that plague the industry. The firm collaborates with hundreds and thousands of photographers all over the world so that they can create images for global brands.

Last year, Meero announced a Series C funding round of nearly 205 million led by Eurazeo Growth and Amsterdam-based VC, Prime Ventures. With this, the valuation of the startup that revolutionised the world of professional photography skyrocketed to 891 million.

Founder/s: Steve Anavi, Alexandre ProtFounded year: 2016Funding: 136 million

Qonto is a neobank for freelancers and SMEs that provides great customer support, transparency, and connection to carry out day-to-day business management. Qonto provides all the necessary tools to master the finances of an organisation. It is designed to make to simple to manage all transfers, debits, card payments, and small details.

Earlier this year, Qonto secured a Series C investment of 104 million from Tencent, Valar Ventures, Alven and DST Global among others, which marks the largest VC round in the French fintech industry to date. With this investment the valuation of the French neobank goes up to 811 million.

Founder/s: Ludovic Le Moan, Christophe FourtetFounded year: 2010Funding: 284 million

Sigfox works with a vision to connect every object in the physical world with the digital universe. It is a platform that provides wireless connectivity services for the IoT devices. The global network of Sigfox complements existing connectivity systems and paves the way for two-way, energy-efficient transmission of small quantities of data over long distances.

Back in 2017, Sigfox announced the closure of an undisclosed amount of funding from Khazanah Nasional Berhad to enhance its deployment in the Asian markets. This venture funding round took its valuation to 600 million.

Founder/s: Acher Criou, Emmanuel Freund, Asher Kagan, Stphane HliotFounded year: 2015Funding: NA

Paris-based Blade is the developer of a dematerialised computer intended to offer cloud gaming servers required to play high-end games through any PC. The companys servers are exploitable on any screen and offer access to a virtual high-end computer along with running thousands of virtual machines on server-grade Intel Xeon processors enabling users to get more power and reactivity.

Earlier this year, Blade, which revolutionises cloud computing, raised funding from LG Electronics. The amount that it raised remains undisclosed but it has secured a position among the most valued French tech startups with a valuation of 335 million.

Founder/s: Charles Gorintin, Jean-Charles SamuelianFounded year: 2016Funding: 125 million

Paris-based digital health insurance platform Alan offers hassle-free digital health insurance solutions to users with an excellent price-quality ratio health plan. This digital insurance platform makes coverage, claims, and reimbursements for both users and medical professionals. Alan also lets users access video calls and appointments.

A few months back, Alan secured 50 million Series C funding from Index Ventures and Temasek. With the latest investment, Alans valuation stands at 314 million and its total funding is 125 million.

Founder/s: Jean-Philippe COMBAL, Gloria Gonzalez-AseguinolazaFounded year: 2016Funding: 84.3 million

Paris-based gene therapy biotech company Vivet Therapeutics is dedicated to developing treatments for liver disorders that have been inherited. The company uses its proprietary treatment for Wilson disease, which is a rare, devastating, chronic and potentially life-threatening liver disorder. This company is building a diversified gene therapy pipeline based on novel adeno-associated virus (AAV) technologies.

In March 2019, Vivet Therapeutics bagged 45 million investment from Pfizer to advance its pipeline of gene therapy programs. This takes the overall funding secured by the French biotech firm to nearly 84 million and its valuation is around 300 million.

Founder/s: David Durrleman, Eric Sibony, Jeremy JawishFounded year: 2014Funding: 89.2 million

Shift Technology is a Paris-based startup providing AI-based fraud detection for the global insurance industry. It offers a fraud detection solution called FORCE, which is used by insurers all over the world across all business sectors. This French AI startup provides fraud handlers with a decision-making platform that will scale their capacity and give them more efficiency in claims processing.

Last year, Shift Technology secured 53 million Series C funding led by Bessemer Venture Partners along with participation from its existing investors such as General Catalyst, Accel and Iris Capital. With this, the valuation of the company is now 277 million.

Main image picture credits: BlaBlaCar

Check out the innovations that took home the Blue Tulip Awards this 2020

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These are the most valued tech startups in France in 2020 - Silicon Canals

What’s a life worth in dollars and cents? Should that influence who gets treated for expensive disease treatments? – Genetic Literacy Project

Austin was three years old and Max was a newborn when their mother, Jenn McNary, learned they had a rare genetic condition called Duchenne muscular dystrophy. The doctor painted a grim picture: Her boys would stop walking by age 12 or 13 and, shortly thereafter, they would require nighttime ventilation. They would each need a tracheotomy, a feeding tube, or both by their late teens. Death would come a few years later.

It hasnt worked out that way, thanks to two new drugs that became available after the boys 2002 diagnosis. Exondys 51, a medicine that targets their genetic mutation, slows the diseases progression, and Emflaza, a corticosteroid, mitigates some of its symptoms. Thanks to these treatments, Austin now attends college and interns at a biotech company. Max attends his local high school in Newton, Massachusetts. Both are able to get around in wheelchairs, and neither needs ventilation. McNary just rented an apartment for her boys because they can function on their own with the help of an aide.

By all accounts, the drugs have been transformative, McNary said. But, she added, her boys arent going to be cured, and extending and improving their life for an unknown period of time comes at a high price. Emflaza came onto the market in 2017 at an annual cost of $65,000. Exondys 51 appeared in 2016 at $748,500. Neither of the drugs will help the young men walk again and, in the eyes of some U.S. health economists, the drugs are not worth the price.

Thats why McNary hates the quality-adjusted life year (QALY, pronounced qua-lee), an economic calculation that attempts to quantify the value of a medical intervention, based in part on the quality of life it bestows on recipients.

First developed by U.S. economists in the late 1960s and early 1970s, variations of the QALY have been used for years by governments around the world to help determine what treatments citizens can obtain under public health care. In Americas free-market health care system, however, QALY calculations have largely been avoided. As McNary and others like her are finding out, thats starting to change.

As policymakers and insurance companies scramble to get a handle on skyrocketing health care costs, they are promoting the idea of paying for value. In this view, drugs designated as higher-value should be prioritized over lower-value treatments. But this raises a thorny question: Who gets to define value? Health economists and insurance companies who seek to use limited health care dollars judiciously? Or patients, parents, and doctors who want to receive the best health care for their situation?

Because the quality-adjusted life year threatens her sons ability to get the medicine they need, McNary is clear about her answer. To me, the QALY is a measurement that says that keeping my sons alive by providing incremental benefit but not totally curing them is never going to be valuable, McNary said. Just mull that around in your head if you are less than perfect, you are worth less money.

In QALY math, a year of perfect health is equal to 1; death equates to 0. The value of other health states is derived from surveys of patients, caregivers, or the general public. Paralysis might be valued at .35, for example, and mild Alzheimers disease at .52, depending on the survey. Those numbers can then be plugged into a formula that allows the relative cost-effectiveness of treatments to be compared to identify the best buys.

Economists developed the QALY concept more than 40 years ago to address a fundamental question: Where should we spend whose money to undertake what programs to save which lives with what probability? Richard J. Zeckhauser and Donald Shepard asked in a 1976 article describing the basic QALY formula. The next year, as U.S. health care spending topped $120 billion, Harvard health policy professor Milton C. Weinstein and his colleague, cardiologist William B. Stason, sounded an alarm bell. It is now almost universally believed that the resources available to meet the demands for health care are limited, they wrote in the New England Journal of Medicine. We, as a nation, will have to think very carefully about how to allocate the resources we are willing to make available for health care.

Their article cited by other authors more than a thousand times in the past four decades pointed out that resources were already being allocated by millions of individual decisions: hospitals rationing beds where they didnt have room for all patients, for example, and insurers agreeing to pay for some tests and treatments but not for others. Such decisions, they argued, were often inconsistent with the societal objective of deriving the maximum health benefits from the dollars spent, an objective that could be achieved by putting the QALY to work.

In the intervening decades, some countries the United Kingdom, the Netherlands, and Sweden, for example have embraced QALY-based evaluations. In the U.K., cost-effectiveness studies are used, in part, to determine which therapies the National Health Service will provide for residents. The publicly-funded health system does not cover Orkambi, the first cystic fibrosis treatment that targets the cause of the disease, for example, because its cost-per-QALY far exceeds the U.K. cost-effectiveness threshold.

In the United States, however, QALY-based assessments have not gained traction until recently. Perhaps the general reason is that we as patients and our providers dont want to be limited in the treatment options available, said Louis P. Garrison Jr., an economist in the Pharmaceutical Outcomes Research and Policy Program at the University of Washington.

In fact, QALY-based cost-effectiveness reviews are so controversial that the federal government has repeatedly quashed their use. In 1992, the Department of Health and Human Services rejected Oregons attempt to use QALY-based cost-effectiveness assessments to determine what services its Medicaid program would cover. In 2010, as part of the Patient Protection and Affordable Care Act, Congress prohibited the use of QALYs by the Medicare program. It also banned the federal Patient-Centered Outcomes Research Institute from using QALY thresholds in its assessments of comparative treatments.

But more than half of U.S. residents are covered by private insurance companies, which are not prohibited from using QALY-based assessments to decide which medicines they will cover for their members. Traditionally, however, private insurers have generally not used QALYs explicitly in their decisions about what tests and treatments they will pay for, according to a recent report by the National Council on Disability. Instead, when major U.S. insurers decide to limit access to a given medication, they usually cite insufficient data to justify its use in a given situation.

Indeed, until recently, U.S. insurers did not have a source for QALY-based cost-effectiveness reports. That began to change in 2014, when the Institute for Clinical and Economic Review, a nonprofit research organization based in Boston, turned its attention to high-cost drugs. Founded in 2006 as a research project based at Harvard Medical School, ICER initially issued reports on broad topics such as obesity management and palliative care. But when Sovaldi, a drug for deadly hepatitis C, came on the market at the then-shocking price of $84,000 for a 12-week course of treatment, ICER kicked into action. Despite the high price, its assessment found that Sovaldi is cost-effective for some patients. Insurers took notice.

Since then, the organization has been churning out several drug-assessment reports each year. Each report includes its opinion of how much the drug is worth; drugs priced higher than that are deemed not cost-effective. ICER has no authority over anyone, but its reports have become popular reading for U.S. insurers. If there is a drug of note being approved by the FDA, theres also likely going to be an ICER assessment of that drug that can factor into their decision-making, said David Whitrap, the research organizations vice president of communications and outreach.

U.S. health care spending has risen dramatically since Weinstein and Stason expressed concern in the mid-1970s. In 2016, the U.S. spent nearly 18 percent of its gross domestic product on health care, far outstripping the average of 11 percent for 10 other high-income nations. High prices for prescription drugs is one reason. Were seeing price tags now of $1 million, $2 million, said Seema Verma, administrator for the federal Centers for Medicare and Medicaid Services, at a conference recently. Thats completely unsustainable for the system.

Thats why Peter Neumann, director of the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center, said cost-effectiveness analyses are needed more than ever. But there are many reasons for the resistance, Neumann and his co-authors wrote in the Journal of the American Medical Association, including an inclination on the part of many individuals in the United States to minimize the underlying problem of resource scarcity and the consequent need to explicitly ration care.

Further, Ari Neeman, a disability rights activist and consultant to Partnership to Improve Patient Care, a coalition of advocacy groups, said the idea that two health conditions can be numerically compared to one another is simply wrong. Proponents of the QALY will say it is this mathematically perfect measure that gives us a superpower ability to compare depression drugs to cystic fibrosis drugs to cancer drugs even though all of those drugs do different things because it lets you translate them back to this common measure, he said. Our concern is that when you engage in that process of translation, you lose some significant nuance in terms of the amount of benefit thats being delivered.

The Partnership argues the QALY calculation is flawed because it assumes quality of life can be captured by a certain number, despite the fact that different surveys arrive at different numbers. For example, a 2006 quality-of-life survey in the U.S. assigns blindness/low vision as .69 on the 0-to-1 scale, while a 2011 survey in the U.K. gives blindness/low vision a score of .78.

Beyond the methodological issues, Neeman said, there are all kinds of ethical problems with it. People with disabilities and chronic medical conditions may value a treatment that offers an incremental improvement in the quality or length of their lives, even though the QALYs gained are less than those for a treatment that prevents the loss of perfect health.

Former U.S. Representative Tony Coelho, a Democrat from California and a primary author of the Americans with Disabilities Act, is the Partnerships chairman. I worry that more focus is being given to what is most cost-effective for the average patient than creating a system that works for each individual patient, he wrote in 2018. The medication I take for epilepsy isnt high value for every patient. But its the only one that works for me.

Thats why, Neeman said, cost-effectiveness analyses must consider the fact that not all patients respond the same way to a drug. Some patients need drugs that arent deemed cost-effective for the general population. Its important to account for that, he said. Otherwise were giving insurers a tool to deny care to people who need it.

When an insurer decides to cover a specific drug, that decision affects everybody who pays into the insurance pool. Michael Sherman, chief medical officer for the insurer Harvard Pilgrim Health Care, uses the example of a gene therapy that costs $1 million to treat a child who will die without it. Under the ACA, families will hit their out-of-pocket maximum at about $16,000, and many health plans have out-of-pocket maximums far below that. The rest of that million dollars is going to be paid by everyone else thats the way it works in insurance, he said. When insurers see that kind of unanticipated budget impact, they raise premiums or out-of-pocket cost-sharing for everyone.

Like other proponents of the QALY, Neumann sees it as an imperfect but useful tool. Any single number is never going to capture everything, he said.

The problem is, if youre not going to use QALYs, what are you going to use?

Thats an urgent question, particularly now when there is a huge pipeline for rare-disease therapies, often called orphan drugs. By 2024, orphan drug sales are expected to reach $242 billion.

In the U.S., a rare disease is defined as one that affects fewer than 200,000 people. While these conditions are individually rare, in the aggregate, an estimated 25 to 30 million Americans thats about one in 10 live with a rare disease. Most rare diseases affect children, and many are fatal or disabling.

Historically, drugmakers spent little effort developing treatments for rare diseases, but that changed with the passage of the Orphan Drug Act of 1983, which provides tax credits and a seven-year marketing exclusivity to companies that develop rare-disease treatments. Hundreds of such treatments have won FDA approval in recent years, with more than 560 medicines in the works.

Those treatments are generally expensive. On average, the per-patient cost for orphan drugs in the U.S. is almost 4.5 times more than for non-orphan drugs.

In the two decades ending in 2017, the average annual cost for orphan drugs was $123,543, based on the price at the time the drug launched, compared to $4,961 for traditional drugs. For Duchenne alone, more than 30 orphan therapies are in development. None of them are going to cure patients, McNary said. But she hopes new treatments, generally used in combination, will help her sons live longer, healthier lives and completely change the disease trajectory for younger patients whose disease has not yet progressed as far.

The barrier she worries about is cost-effectiveness analysis. In August, the Institute for Clinical and Economic Review published its assessment of treatments for Duchenne, which affects about 400 to 600 boys born in the U.S. each year. Emflaza, the corticosteroid, appears to be as good as or better than prednisone, another corticosteroid approved to treat the disease, but it would need a price cut of at least 73 percent to be considered cost-effective.

Exondys 51 approved by the FDA for about 13 percent of the Duchenne population got a worse review. In the clinical trials used to seek FDA approval, no clinical benefit, including motor function improvement, was demonstrated. (The FDA approved the drug because some of the patients treated with Exondys 51 had a slight increase in dystrophin levels in skeletal muscle.) In light of that, Exondys 51 was not deemed cost-effective at any price.

But Jenn McNary said the drug works for her sons. Austin, who was not eligible for the Exondys 51 clinical trial, stopped walking at age 10. Max got in the trial and started taking the drug at age 9.They have the same mutation, they have been raised by the same mother, so one would expect they would progress similarly, she said. But Max walked until he was 17.

Austin was already in a wheelchair when, at age 15, he started taking Exondys 51. He regained some upper-body strength that changed his life, according to his mother. Hes able to use a urinal on his own, which makes is possible for him to have a job and to go to college without an aide, she said.

The Medicaid program in Massachusetts, where the McNarys live, wont pay for Maxs Duchenne therapies. For the time-being, the drugmakers are giving him the drugs free through a patient-assistance program. Austin, because hes enrolled in college, is eligible for student coverage through Blue Cross Blue Shield of Massachusetts. The insurer, by policy, does not cover Exondys 51 for patients who can no longer walk. His mother appeals the insurance denial. Every six months, she sends a video of Austin in action, along with a letter from his doctor and so far, his medicines have been covered.

The payers made their coverage policies before the quality-adjusted life year analysis was published. Now, insurers who have been covering the Duchenne treatments have an independent analysis with which to rethink that decision.

For now, there is one thing that QALY supporters and critics agree on. Very promising drugs are coming, and theyre going to be very expensive, said Neumann, the health economist at Tufts. Increasingly, the QALY appears poised to influence how American health care money is spent.

Lola Butcher is a health care business and policy writer based in Missouri. Follow her on Twitter @LolaButcher

This article was originally published on Undark and has been republished here with permission. Undark can be found on Twitter @undarkmag

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What's a life worth in dollars and cents? Should that influence who gets treated for expensive disease treatments? - Genetic Literacy Project

FDA Approves Genentech’s Phesgo (Fixed-dose Combination of Perjeta and Herceptin for Subcutaneous Injection) for HER2-positive Breast Cancer -…

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, Inc., a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has approved Phesgo, a fixed-dose combination (FDC) of Perjeta (pertuzumab) and Herceptin (trastuzumab) with hyaluronidase, administered by subcutaneous (SC, under the skin) injection in combination with intravenous (IV) chemotherapy, for the treatment of early and metastatic HER2-positive breast cancer. This is the first time that Genentech has combined two monoclonal antibodies that can be administered by a single SC injection.

The FDA approval of Phesgo reflects our commitment to improving outcomes for the many people living with HER2-positive breast cancer, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. Phesgo offers a treatment administration that supports the needs and preferences of individual patients, and helps to meet the increasing demand across the healthcare system for faster and more flexible treatment options.

Phesgo is available in one single-dose vial. Administration can take approximately eight minutes for the initial loading dose and approximately five minutes for each subsequent maintenance dose. This is compared to approximately 150 minutes for infusion of a loading dose of Perjeta and Herceptin using the standard IV formulations, and between 60-150 minutes for subsequent maintenance infusions of the two medicines. Phesgo can be administered by a healthcare professional in a treatment center or at a patients home.

The approval is based on results from the pivotal Phase III FeDeriCa study, which met its primary endpoint with Phesgo showing non-inferior levels of Perjeta in the blood during a given dosing interval (Ctrough) when compared to IV administration of Perjeta. The safety profile of Phesgo with chemotherapy was comparable to IV administration of Perjeta plus Herceptin and chemotherapy, and no new safety signals were identified, including no meaningful difference in cardiac toxicity. The most common adverse events in both arms were alopecia, nausea, diarrhea and anemia.

The Phase II PHranceSCa study showed that 85% (136/160) of people receiving treatment for HER2-positive breast cancer preferred treatment under the skin to IV administration due to less time in the clinic and more comfortable treatment administration.

For those who qualify, Genentech will offer patient assistance programs for people prescribed Phesgo by their doctor through Genentech Access Solutions. Please contact Genentech Access Solutions at (866) 422-2377 or visit for more information.

About the FeDeriCa study

FeDeriCa is an international, multi-center, two-arm, randomized, open-label, Phase III study evaluating the pharmacokinetics, efficacy, and safety of SC injection of Phesgo in combination with chemotherapy, compared with standard IV infusion of Perjeta and Herceptin in combination with chemotherapy, in 500 people with HER2-positive EBC who are being treated in the neoadjuvant (before surgery) and adjuvant (after surgery) settings. The primary endpoint of the study is minimum levels of Perjeta in the blood during a given dosing interval (Ctrough). Secondary endpoints include safety; minimum levels of Herceptin in the blood during a given dosing interval (Ctrough); and total pathological complete response, meaning there is no tumor tissue detectable in the tissue removed at the time of surgery. The safety profile of Phesgo was comparable with that of Perjeta and Herceptin administered intravenously.

Data from the FeDeriCa study were presented at the San Antonio Breast Cancer Symposium in December 2019. The FeDeriCa study met its primary endpoint of non-inferior levels of Perjeta in the blood. The geometric mean ratio (GMR; a type of average used when assessing pharmacokinetics) for the primary endpoint was 1.22 (90% CI: 1.14 to 1.31), with the lower limit of the 90% CI of the GMR=1.140.80 (the pre-specified non-inferiority margin). A secondary endpoint of non-inferior levels of Herceptin was also met, with blood concentrations for people receiving the fixed-dose combination non-inferior to those receiving IV Herceptin (GMR=1.33 [90% CI: 1.24 to 1.43]; lower limit of 90% CI of GMR=1.240.80). A non-inferiority endpoint was chosen for the study to ensure that people were receiving sufficient dosing with Perjeta and Herceptin as compared to the established IV doses at the same treatment intervals.

About the PHranceSCa study

PHranceSCa is a randomized, multi-center, multinational, open-label, cross-over Phase II study evaluating patient preference for and satisfaction with subcutaneous (SC) administration of Phesgo. All patients completed neoadjuvant treatment with Perjeta, Herceptin and chemotherapy and had surgery before randomization. The primary endpoint of the study is the percentage of participants who indicate that they prefer treatment with Phesgo compared to the standard intravenous (IV) formulations of Perjeta and Herceptin. Secondary endpoints include participant-reported satisfaction and health-related quality of life outcomes; healthcare professionals' perceptions of time and resource use and convenience compared with IV formulations; as well as the safety and efficacy of each study regimen.

About HER2-positive breast cancer

Breast cancer is one of the most common cancers among women worldwide. According to the American Cancer Society, close to 280,000 people in the United States will be diagnosed with breast cancer, and more than 42,000 will die from the disease in 2020. Breast cancer is not one, but many diseases based on the biology of each tumor. In HER2-positive breast cancer, there is excess HER2 protein on the surface of tumor cells. Approximately 15-20% of breast cancers are HER2-positive based on the result of a diagnostic test.

About Phesgo

Phesgo (subcutaneous Perjeta and Herceptin) is a new fixed-dose formulation of Perjeta and Herceptin with Halozyme Therapeutics Enhanze drug delivery technology.

Trastuzumab in Phesgo is the same monoclonal antibody as in IV Herceptin and pertuzumab in Phesgo is the same monoclonal antibody as in IV Perjeta. The mechanisms of action of Perjeta and Herceptin are believed to complement each other, as both bind to the HER2 receptor, but to different places. The combination of Perjeta and Herceptin is thought to provide a more comprehensive, dual blockade of HER signaling pathways, thus preventing tumor cell growth and survival.

Halozymes Enhanze drug delivery technology may enable and optimize SC drug delivery for appropriate co-administered therapeutics. The technology is based on a proprietary recombinant human hyaluronidase PH20 (rHuPH20), an enzyme that temporarily degrades hyaluronan a glycosaminoglycan or chain of natural sugars in the body, to aid in the dispersion and absorption of other injected therapeutic drugs.

Phesgo Indications and Important Safety Information

Phesgo (pertuzumab, trastuzumab, and hyaluronidase-zzxf) is a prescription medicine approved for use in combination with chemotherapy for:

Phesgo is also approved for use in combination with docetaxel in adults who have HER2-positive breast cancer that has spread to different parts of the body (metastatic) and who have not received anti-HER2 therapy or chemotherapy for metastatic breast cancer.

Important Safety Information

What should patients know about side effects with Phesgo?

Most serious side effects with Phesgo

Phesgo may cause heart problems, including those without symptoms (such as reduced heart function) and those with symptoms (such as congestive heart failure).

Receiving Phesgo during pregnancy can result in the death of an unborn baby and birth defects.

Phesgo may cause serious lung problems.

Who should not receive Phesgo?

Other possible serious side effects

Most common side effects

The most common side effects of Phesgo when given with chemotherapy as part of an early breast cancer regimen are:

The most common side effects of Phesgo when given with docetaxel for treatment of breast cancer that has spread to other parts of the body (metastatic) are:

Patients are encouraged to report side effects to Genentech and the FDA. Report side effects to the FDA at 1-800-FDA-1088 or Report side effects to Genentech at 1-888-835-2555.

Patients should talk to a healthcare professional for more information about the benefits and risks of Phesgo.

Please see full Prescribing Information for additional Important Safety Information, including most serious side effects.

If a patient cannot afford their medication, visit for financial assistance information.

About Expanded Access Treatment Protocol for Continuity of Care During COVID-19

Genentech launched an expanded access treatment protocol in the United States, where the FDC of Perjeta and Herceptin is administered at home by a home health nursing provider. The study will continue beyond approval, aiming to help continuity of care during the COVID-19 pandemic for certain patients with HER2-positive breast cancer who have completed chemotherapy concurrent with Perjeta and Herceptin intravenously and are currently receiving or will be receiving Perjeta and Herceptin alone. To learn more, please visit here.

About Genentech Access Solutions

Access Solutions is part of Genentechs commitment to helping people access the Genentech medicines they are prescribed, regardless of their ability to pay. The team of in-house specialists at Access Solutions is dedicated to helping people navigate the access and reimbursement process, and to providing assistance to eligible patients in the United States who are uninsured or cannot afford the out-of-pocket costs for their medicine. To date, the team has helped more than 2 million patients access the medicines they need. Please contact Access Solutions (866) 4ACCESS/(866) 422-2377 or visit for more information.

About Genentech in breast cancer

Genentech has been advancing breast cancer research for more than 30 years with the goal of helping as many people with the disease as possible. Our medicines, along with companion diagnostic tests, have substantially improved outcomes for HER2-positive breast cancer. As our understanding of breast cancer biology rapidly improves, we are working to identify new biomarkers and approaches to treatment for other subtypes of the disease, including triple-negative and hormone receptor-positive.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit

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FDA Approves Genentech's Phesgo (Fixed-dose Combination of Perjeta and Herceptin for Subcutaneous Injection) for HER2-positive Breast Cancer -...

Conversations Between Patient and Oncologist Are Important in Avoiding ‘Common Mistake’ When Treating CLL –

In an interview with CURE, Dr. Mazyar Shadman discussed the enormous variety of treatment preferences among patients with CLL and said that emphasis should be placed on treating each person according to their wishes.

BY Ryan McDonald and Dr. Mazyar Shadman

A common mistake that tends to occur when patients are treated for chronic lymphocytic leukemia is the notion that only a patients cells are being treated, according to Dr. Mazyar Shadman.

In an interview with CURE, Shadman, of Seattle Cancer Care Alliance and Fred Hutchinson Cancer Center, discussed the enormous variety in treatment preference among patients with CLL and said that emphasis should be placed on treating each person according to their wishes.


The main bulk of my discussion is really what they want. You will be surprised at how different patients are in terms of what's important for them. One side of the spectrum, I have patients who really don't want a treatment that's forever, and that makes sense, right? No matter how safe the drug is, no matter how disease-free you are, just for some patients, just the thought of being on medication forever that's for cancer is important. And they just don't want it; they want to be disease-free, but also treatment-free as long as possible. And so that's important for some patients, and in the appropriate setting, if I feel that, yes, you know, we can go with either chemotherapy-based therapy in this patient or now venetoclax-based therapy (which) is a non-chemotherapy and a time-limited option potentially. For those patients based on that decision, of course, that's what I will provide to them.

I also have patients who really don't (care, and will say) I'm taking four medications for years, four different products, and (if) you give me something, I don't want to deal with the whole rampup and checking MRD, (but) as long as I'm fine,, I'll take it, so that you see both sides, and you do see patients who still come for chemotherapy. So, yes, patient preference is important. Patients need to understand and need to be educated on what to expect. It's always easy to start treatment. We get patients who come back six months, seven months later, and (say) You know what, I'm taking this for seven months it works, but can I stop? I mean, what do you mean (this) is going to go forever? The doctor who started didn't talk to me about (that).

So, I think those conversations are actually very important. And I add one more thing, they're important because we don't have a head-to-head trial that tells me that acalabrutinib or venetoclax which one is better. If I have that, then I probably won't give that option, or if I do, I make sure they understand that they're going for a treatment that's not superior, but we don't have that. So really, you have to sit and present both treatments and pros and cons and have them decide based on what's the best fit for them. And that's why it's important to have really a comprehensive approach to the patient I mean, it's really treating a patient, not the molecular kind of finding in the CLL cell, and that's a common mistake: Oh, I have somebody with a mutated (gene, and) that (means) I give chemo. No: You have a patient who has CLL whose CLL has this (mutation). So, then you kind of look at the patient and not the other factors.

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Conversations Between Patient and Oncologist Are Important in Avoiding 'Common Mistake' When Treating CLL -

Drug used in breast and ovarian cancer could lead to safer, more effective treatment for neuroblastoma – The Institute of Cancer Research

Image: Neuroblastoma rosettes. Credit:Dr. Maria Tsokos, National Cancer Institute(Public domain).

A type of drug known as a PARP inhibitor, already used to treat breast and ovarian cancer, may be useful in treating children with high-risk neuroblastoma a common childhood tumourwith a low survival rate.

Neuroblastoma is a cancer in childrenwhich arises from nerve cells in either the chest or the abdomen. In some neuroblastoma cases there is an abnormal gene called MYCN that makes it particularly difficult to treat.

Currently, children receive intense chemotherapy, radiotherapy and surgery but despite this only about half of children with high-risk neuroblastoma will survive.

Researchers at Sheffield Universitycollaborated with The Institute of Cancer Research, London, to conduct research to find a new, more effective and less toxic way to target high-risk neuroblastoma tumours with increased levels of MYCN.

MYCN affects how tumour cells grow and makes them more dependent on a protein called PARP for survival. This means that the tumour cells are specifically killed by a drug that stops PARP working. An advantage to this type of therapy is that normal non-tumour cells are not harmed so the damaging side-effects often seen with chemotherapy are reduced.

The study, funded by childhood cancer research charity Children with Cancer UK, was led by Dr Helen Bryant and Dr David King in Sheffield, in collaboration with Professor Louis Chesler at The Institute of Cancer Research (ICR).

They found that the use of PARP inhibitors alone, and alongside chemotherapy, was effective in killing neuroblastoma cells, leaving behind healthy cells. This is one of the first studies to show this in neuroblastoma cells and in tumour models.

Dr David King, childrens doctor at Sheffield Children's NHS Foundation Trust and Children with Cancer UK researcher, said:

Children with neuroblastoma and MYCN currently receive some of the most intense treatment used for any type of cancer. The harsh treatment causes numerous side effects and children often have long hospital stays. And despite the treatment, only about half of children with high-risk neuroblastoma will survive. PARP inhibitors are known to have very few side effects in adults and we have shown they may be an effective treatment for neuroblastoma. Their use in neuroblastoma could mean more children survive the disease and need less toxic treatment.

We have shown that PARP inhibitors kill neuroblastoma cells with MYCN in the laboratory. The next step is to see whether they are beneficial in children with neuroblastoma, probably in combination with chemotherapy or radiotherapy. This would be done in a clinical trial.

The ICR is an internationally leading research centre in the study of childhood cancers and cancers in children, teenagers and young adults.

Find out more

Dr Helen Bryant, research group lead and senior lecturer at Sheffield University, said:

Our study has examined the way DNA is damaged and repaired one of the features common in all cancers is that there are changes in that process. We were interested in how that process changes in neuroblastoma and whether we can exploit those changes to specifically kill cancer cells and leave the normal cells around the tumour unharmed. In this way we hope to develop kinder and less toxic therapies for children.

PARP inhibitors are being used successfully in women with breast and ovarian cancer so we are optimistic that this can be translated fairly quickly into children with neuroblastoma.

Mark Brider, Chief Executive Officer, Children with Cancer UK, said:

Neuroblastoma is one of the most common childhood tumours with around 100 children, mostly under five years old, diagnosed every year in the UK. Yet it also has one of the lowest survival rates in its high-risk form the survival rate is sadly around just 50%. It remains one of the most difficult childhood cancers to cure.

It is crucial that we find more effective and personalised treatments for children with neuroblastoma. The findings of Dr Bryant, Dr King and the teams in Sheffield and London represent an important step towards the development of new and kinder treatments that reduce the burden of toxicity for young cancer patients and improve survival rates in this aggressive and hard-to-treat cancer.

We are building a new state-of-the-art drug discovery centre to create more and better drugs for cancer patients. The centre is a 75m project and we now have less than 2m to raise. Help us finish and equip the building to get our research off to the best possible start.

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Alice Latham, 3, from Derbyshire, was diagnosed with high-risk neuroblastoma, with the MYCN gene, a week before her second birthday in January 2019. Her parents, Jamie and Helena, explain the harmful side-effects of Alices treatment.

Alices neuroblastoma treatment has been very aggressive and harsh on her body, she has experienced many side effects, including hair and weight loss. The treatment may also have an impact on her later in life she may not grow to her full height and there is a chance she may not be able to carry children. If treatments could be developed that are less harmful to childrens growing bodies that would be incredible, said Helena.

Jamie added:

As a parent you know nothing about neuroblastoma you understand when you get the diagnosis that the prognosis for the high-risk form is 40-50% survival after all of the treatment. The research for this type of cancer has really lagged behind and the treatment is very hard hitting. It is vital for charities like Children with Cancer UK step in and continue to fund this lifesaving research.

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Drug used in breast and ovarian cancer could lead to safer, more effective treatment for neuroblastoma - The Institute of Cancer Research

Family Of Dying Santa Clarita Teen Pleads With Community For Donations To Fund Life-Saving Treatment – KHTS Radio

The family of a Santa Clarita teen with Batten disease, a fatal degenerative genetic disorder, is pleading with the community for help after suddenly learning that scheduled life-saving research had been halted, and is now being faced with an urgent need for additional fundraising support.

Affectionately known as Sebi, Sebastian Velona was born in 2003, and was a perfectly happy, healthy young boy with a big heart for the first several years of his life, according to his mother, Teri Hughes Fox.

He would also have the biggest heart and just want to hug everyone, Teri said. He just felt like, if you had a boo-boo, if you had a scrape, if you were crying, if youre hurt, if youre sad He felt he could hug people and heal them with his hugs.

Then, in October of 2007, four-year-old Sebi and his family were on vacation in Hawaii when tragedy struck.

He collapsed in the hallway going back to the room after having a fun day swimming in the pool, Teri said. He collapsed and had his first seizure. We thought he was dying. We didnt know what was wrong. He was turning blue. He was shaking. It was just horrible.

When the family returned home, a series of tests were unable to find any cause for Sebastians seizure, and a neurologist deemed it a one-off fever seizure.

Six months later, he had a second seizure. At that point, Sebi was diagnosed with epilepsy.

That was just the worst word at that time Id ever heard, Teri said. Our world changed.

After that came years of trial and error with medications, some of which came with side effects that further endangered Sebis health. Some medications resulted in mood swings and outbursts of anger, where others caused him to suffer more than 10 seizures in a day.

Eventually, Sebis doctors discovered a cocktail of three medications that managed to control his seizures, but the problems were just beginning.

When he was six, Sebi began to lose his vision, eventually being diagnosed with retinitis pigmentosa, a degenerative eye disease that will eventually cause him to lose his sight entirely.

He was at school learning to read and all of a sudden, overnight, it seemed like he couldnt see what was on the page, Teri said. It was fuzzy, he couldnt read it. There was something wrong.

It was at this time that Sebis family began to notice other issues developing in his behavior. He became clumsier and his speech began to slur. His parents insisted that there was something else at work, and obtained a DNA test to narrow down the cause of Sebis health issues in order to get him treatment.

Im thinking: Its not just retinitis pigmentosa and epilepsy. Theyre not separate issues where my son is just developing all these horrific diseases, Teri said. Theres something else wrong, everything about him is different, everything is changing and unless a doctor sees you every day, they dont see what were talking about.

After months of waiting, Sebi was diagnosed with CLN8 Batten disease in 2012. Batten disease is a disorder that primarily affects the nervous system, leading to neurological impairment, including developmental regression, seizures, blindness, behavior changes and dementia, according to health officials.

Teri described Batten disease as similar to a juvenile form of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrigs disease. The two conditions share many of the same symptoms.

The condition occurs in an estimated two-to four-in-100,000 live births in the United States, according to the National Institute of Neurological Disorders and Stroke (NINDS).

His parents immediately set to work to advocate on behalf of their child, working hard to raise the $3.5 million that would be needed in order to fund life-saving gene therapy research for Sebi and other children with CLN8. They founded the Sebastian Velona Foundation (SVF) to help raise the necessary funds.

Over the past several years, the family has advocated for gene therapy for their son after discovering successful forms of gene therapy to battle CLN6, another form of the disease.

In 2018, Senator Scott Wilk, R-Santa Clarita, sponsored a bill that designated the first weekend in June as Batten Disease Awareness Week. Wilk was made aware of the deadly disease and the need for more awareness after Chris Velona, Sebis father, traveled to Sacramento and sought out support for his son, as well as the thousands of other children and their families who have been affected by the disease.

Unfortunately, due to Food and Drug Administration (FDA) guidelines and other safety regulations, it has taken more than three years to reach the point where gene therapy research is able to take place. The SVF partnered with Columbus, Ohio-based Nationwide Childrens Hospital, which had placed gene therapy on the map according to Teri.

Nationwides Childrens Hospitals gene therapy program was bought by another company, before eventually being acquired by Amicus Therapeutics approximately 18 months ago.

Unfortunately for us because of the regulations, and the changing of the companies and the acquisitions, were kind of confused as to where were at, Teri said. Its really slowed us down.

The gene therapy is now in the process of being transferred back to Nationwide Childrens Hospital in order to complete the process and bring the result to human trials as quickly as possible.

Although Nationwide Childrens Hospital is more than willing to perform the research, they simply do not have the budget available to fund the project. The funding for the final stretch of research instead falls to the Velona family, with a price of at least $500,000.

We need to raise $500,000 and we need to do it yesterday, Teri said. We need to do this now and get the word out there, that there are so many with Batten disease that we could help with this gene therapy. As the pioneer, its going to get less expensive to bring this treatment to the masses.

According to Teri, the gene therapy treatment can not only halt the progression of the disease, but also save Sebis life as well as the lives of others affected by it. Without treatment, it is highly likely that within a few years at most, his condition will progressively deteriorate until he is unable to speak, see, walk, or even eat, until he would eventually die.

Teri worries that all families can do is wait for funds and treatment, knowing that waiting will only worsen their childs condition and quality of life.

We dont have the time to wait if he gets any worse we dont want to leave him in a state where its not a good quality of life, she said.

According to his mother, as of early June, Sebi is barely able to see, his speech is muddled and often unrecognizable, his cognitive thinking has deteriorated, and his physical and motor skills have so declined that he is unable to write or walk unaided. He is deteriorating on a daily basis, she said.

This is my son, he was fine, it just happened so fast, Teri said. Now were scrambling, kicking down any door, looking for anyone that will hear us, listen to us. We cant just watch our son deteriorate and die. Im not a good mom if I sit there and do nothing.

The news about new funding and the delays in gene therapy comes just as Sebi lost one of his grandfathers to COVID-19 in April, a death that has been devastating for him, according to his mother.

Hes very frustrated and sad. He keeps asking When is the gene therapy? asking me It was going to be soon, where is it? Teri said. Its my job to take care of him and fix him, and I cant fix him. So its hard.

Teri says that her 16-year-old son understands what is happening to him and why he cant go to school or get a drivers license like other teens his age, but he keeps a positive outlook through the difficulties he experiences every day, and even attempts to comfort her whenever the frustration becomes too much to handle.

He has an amazing attitude he wants to help people with his hugs, she said. If he saw me crying right now he would lose it, and be hugging me, and petting me and telling me, Mom its going to be okay, Im going to get gene therapy.

Although his body is failing him, it is Sebis good attitude about the situation that helps motivate his family, according to his mother.

He is so positive in his outlook that he makes me a better mom. Im not allowed to get down, or cry, or get frustrated, or kick and scream, Teri said. Im mad this gene therapy is taking so long. But Im so grateful that we have this opportunity to actually get a treatment

After receiving funding for gene therapy treatment, Teri and the SVF plan to continue helping other children with CLN8 around the world, and to help others with rare diseases.

Everyone deserves a chance, she said. The disease can be stopped by one injection, replacing the good gene with the bad gene. It baffles me.

In a literal race against time, the doctors at National Childrens Hospital and the Velona family remain dedicated to making sure that the gene therapy goes to trial before it is too late to save Sebastian and the other children like him.

Im not going to let my baby go that way, we need to fight, we need to raise money, its worth everything, Teri said. I know there are so many amazing charities out there, but you can actually save some childrens lives right now.

To support gene therapy research for Sebi, or to learn more about the SVF, click here.

KHTS FM 98.1 and AM 1220 is Santa Claritas only local radio station. KHTS mixes in a combination of news, traffic, sports, and features along with your favorite adult contemporary hits. Santa Clarita news and features are delivered throughout the day over our airwaves, on our website and through a variety of social media platforms. Our KHTS national award-winning daily news briefs are now read daily by 34,000+ residents. A vibrant member of the Santa Clarita community, the KHTS broadcast signal reaches all of the Santa Clarita Valley and parts of the high desert communities located in the Antelope Valley. The station streams its talk shows over the web, reaching a potentially worldwide audience. Follow @KHTSRadio on Facebook, Twitter, and Instagram.

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Family Of Dying Santa Clarita Teen Pleads With Community For Donations To Fund Life-Saving Treatment - KHTS Radio

Hungarian Neurobiologist Botond Roska to Receive This Year’s Krber European Science Prize – Hungary Today

Botond Roska, a Hungarian neurobiologist researching in Switzerland, will receive the Krber Foundations prestigious award, the Krber European Science Prize, which recognizes a single European scientist each year, alternating between life sciences and physical sciences. The Hungarian physician, one of the worlds leading experts in the study of vision and the retina, is to be awarded for his work that revolutionised ophthalmology.

According to the website of the Krber Foundation, the Hungarian neurobiologist Botond Roska has set himself the goal of restoring sight to the blind. Roska and his research group in Switzerland investigate how various cell types in the visual system extract visual information from the environment. Based on molecular mechanisms, they have designed novel gene therapies for restoring vision in genetic forms of blindness. The scientist has carried out pioneering work to identify approximately one hundred different cell types in the retina and their complex interplay in signal processing.

The Krber European Science Prize honors outstanding and excellent scientists working in Europe. The prize is awarded to research projects that show great potential for possible application and international impact. In the last ten years alone, the Krber Prize winners included six scientists who were later awarded the Nobel Prize.

Roska is now working on making these fundamental insights beneficial for patients and using gene therapies to alleviate or cure their diseases. A genuine breakthrough was achieved by reprogramming a cell type in the eye, enabling it to take over the function of defective light receptor cells. He was thus able to make blind retinas light-sensitive again and clinical trials with blind people have already begun.

Hungarian-born Researcher Awarded for Helping People Regain Their Sight

In 2019, Roska was awarded the Louis-Jeantet Prize for Medicine for the discovery of basic principles of visual information processing and the development of therapeutic strategies, such as gene therapy, to restore vision in retinal disorders. He is also the first Hungarian to earn the Bressler Prize for his vision-restoring therapy, and the Alden Spencer Prize for understanding the vision process.

Later last year, President Jnos der decorated the neurobiologist with the Hungarian Saint Stephen Order on August 20th, Hungarys national holiday. der referred to Roska as a doctor who does not treat patients but enables others to heal through his research.

August 20 Saint Stephen Order Goes to Outstanding Neurobiologist Botond Roska

The father of the world-renowned researcher was electrical engineer Tams Roska, founder of the research of neural networks in Hungary. Botond Roska, although first studying cello at the Liszt Ferenc Academy of Music in Budapest, had to give up his music career because of an injury. He later received his medical degree from Semmelweis University, a PhD in neurobiology from the University of California, Berkeley, and studied genetics and virology at Harvard University. Roska currently works in Basel, Switzerland as co-director of the Institute of Ophthalmology Basel (IOB).

The Krber European Science Prize 2020 will be presented to Roska on7 September in the Great Festival Hall of Hamburg City Hall.

featured photo: Nomi Bruzk/MTI

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Hungarian Neurobiologist Botond Roska to Receive This Year's Krber European Science Prize - Hungary Today

Quarterly summary: the COVID-19 lockdown and the importance of scientists – University Affairs

The Black Hole

Both David and Jonathan hope that many new and good things can come from this period of lockdown.


This quarter has been different there is no doubt about that. Both Jonathan and I have spent lockdown with working partners (medical doctor and scientist) and two little ones each (all four kiddies under the age of five) and we were chatting the other day about how odd it is that he and I have only ever met physically in person three times. Sometimes people just get on and have aligned goals and ambitions, and The Black Hole has been a tremendous pleasure to share with Jonathan (and Beth Snow before!). This period of enforced online interaction has led to some serious Zoom fatigue, but we both hope that many new and good things can come from this period of lockdown as youll see from the range of posts below.

This quarters most popular posts were my recent post on career considerations during lockdown and Joanthans post from last summer entitled You are not a failure for wanting to leave academia which we imagine also got some extra readers during lockdown for similar reasons as the career considerations post. We hope that readers enjoy these and others. We also hope to write more on the positive things that might emerge from the introspective journey that many of us have been on over the last months (or the one well go on after life gains some semblance of normality!). Our recent posts are listed below:

This quarter Dave has also written some more science-y stuff with a series of articles exploring recent scientific advances in blood stem cell gene therapy for a wide range of diseases over on the Signals blog:

Were looking forward to getting back to normal (even to whatever the new normal will be) and as always, encourage our readers to contribute their own articles to this column to gain some exposure for issues near and dear to their hearts.

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Quarterly summary: the COVID-19 lockdown and the importance of scientists - University Affairs

Flames assistant GM continues to defy the odds in his battle against ALS – CTV News

CALGARY -- After being diagnosed with ALS, Calgary Flames assistant general manager Chris Snow appreciates every minute he gets to spend with his family.

He never takes it for granted.

He also appreciates recent fundraising efforts similar to the Ice Bucket Challenge that have been taking social media by storm, in his honour.

On Monday Snow was tossing the ball around with his children, eight year old Cohen and five year old Willa.

Just over a year ago, he didnt think he was going to be able to do that.

He says hell never forget that day, when a doctor told him he had ALS.

"He looked at me and said this is the early stage and you just sink," he said.

"You think your life is over because it really is. At least until now, in every case, it's a no hope disease."

About 3,000 Canadians currently live with ALS. Its a rapid and always fatal neurodegenerative disease that typically hits those aged between 55 and 65.

Chris is just 38 years old and ALS runs in his family. His dad, two uncles and a cousin all passed away from it.

The Snows have the most aggressive sub-type of the disease. Chris was given just 12 months to live.

But he and his wife Kelsie werent about to give up. Kelcie did the research and Chris was enrolled in a gene therapy clinical trial.

Kelcie says that gave them hope.

You know when I asked the doctor, 'is it possible this could stop their progression entirely?' and he said, 'we dont know but its not outside the realm of possibility,'" she said.

"We held onto that and we keep holding onto that."

Chris says so far its appears to be working.

My right hand, it doesnt really do anything for me. But it hasnt since I enrolled in the trials. I didnt see that as a form of progression because that was already gone," he said.

My upper arm on that side remains very strong. I can lift and hold my kids with that. Just have to catch them right way.

June is ALS awareness month. And Snow and the Flames came up with a unique idea to help raise money to fight the disease.

Its called Trick Shot for Snowy and has taken twitter by storm. Snow says hes been loving all of the creativity.

Its like the ice bucket challenge but its fun," he said. "You get to show off a little bit. And do things that are either skillful or ridiculous. And social media has the ability to reach so many people at this point in time."

So far, Trick Shot for Snowy has raised more than $150,000. Chris says all the money raised will be put toward the best research they can find in Canada.

Kelcie says she couldnt be more proud of her husband.

You know in some ways its like he was made for something like this," she said. "Hes so positive and he always finds a way to get by and to keep going and smiling."

Just over a year ago Snow thought he was given a death sentence. On Sunday, Fathers Day, he hiked a mountain with his family.

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Flames assistant GM continues to defy the odds in his battle against ALS - CTV News

3 Reasons to Go Long Crispr Therapeutics – InvestorPlace

For years, I consistently urged investors to focus the bulk of their portfolios on what I term megatrends. While making a quick buck on momentum plays is great, your highest probabilities of success rest on long-term developments in technology and innovation. And few hit all the right buttons like Crispr Therapeutics (NASDAQ:CRSP). Levered to the groundbreaking gene-editing industry, CRSP stock deserves extra attention for anyone interested in phenomenal gains.

Source: Catalin Rusnac/

Its amazing to think how far technology has come. What was once the exclusive realm of science fiction, we are now closer than ever to a fully automated society. From driverless vehicles to automated customer service platforms to smart city infrastructures, investing in innovation has never been more relevant.

At the same time, medical innovations have somewhat lagged the profound changes were seeing everywhere else. Try as we might, we havent had many breakthroughs regarding the human condition. Primarily, our practically available medical solutions involve mitigating symptoms, not addressing root causes. Crispr Therapeutics hopes to disrupt this paradigm entirely, thereby imbuing CRSP stock with its potential.

Underlining the biotechnology firms awe-striking platform is CRISPR, which is a specialized stretch of DNA. Associated with CRISPR is a protein called Cas9, which essentially acts as a pair of molecular scissors. This function enables the gene-editing process, which from a broad summary view involves cutting problematic gene sequences and correcting them.

One of the most important components of the CRISPR-Cas9 technology is that scientists adapted it from a naturally occurring genome editing system in bacteria. Thus, were not necessarily talking about playing God. Instead, were utilizing breakthroughs in biotechnology to transfer natural processes to aid humanity.

I couldnt think of a better argument for CRSP stock. But here are three more for your consideration.

In the biotech space, the biggest prize is the cure for cancer. By the end of this year, experts predict that 1.8 million Americans will be diagnosed with the disease. Tragically, over 600,000 will succumb to cancer. Thus, its imperative that we marshal all available resources to fight this scourge.

Unfortunately, the mechanisms by which we treat the disease is rather antiquated. Years from now, I can imagine that people will look back and consider our tactics barbaric. Rather than attacking the cancer cells themselves, we address symptoms, mainly through surgery, chemotherapy and radiation therapy.

In other words, cut em, drug em and fry em. That might work if were in the fake meat processing business. But to treat our loved ones, it seems crude and anachronistic.

And this is why Im very excited about the potential of CRSP stock. To be clear, Im not suggesting that Crispr is on the cusp of a cancer breakthrough. However, with their innovative approach modifying a patients immune cells to efficiently attack cancer cells the possibilities are simply astonishing.

Currently, the major knock against this platform is its extreme cost, which brings up ethical concerns. However, through industry partnerships and synergies, its likely that over time, these costs will come down, making them a more affordable choice for patients.

One of the worst pieces of bad news you can get from the doctor is a diagnosis of a rare disease. Unlike other conditions, such as cancer, support networks may be limited in number. In addition, the treatment options may be few as well. Plus, the available solutions are typically very expensive.

But the most difficult challenge is the helplessness that patients suffer. For instance, with lupus, the body basically attacks itself. Traditional medicines have focused on symptom mitigation. But what can you do when your body stands divided?

Its precisely this type of condition for which Crispr provides meaningful hope. With the companys gene-editing technology, it can potentially alter the lives of millions who suffer silently.

As with cancer, Crisprs current mechanism isnt cheap and therefore, isnt practical for all but the wealthiest patients. Again, though, advancing technologies always drive down costs. And frankly, having the foundation for such groundbreaking solutions is far better ethically than not having it whatsoever.

Therefore, I believe investors can buy into CRSP stock with a clear conscience.

Easily the most powerful yet controversial implication of the CRISPR-Cas9 technology is the development of gene drives. According to Live Science contributor Aparna Vidyasagar, gene drives are:

genetic systems, which increase the chances of a particular trait passing on from parent to offspring. Eventually, over the course of generations, the trait spreads through entire populations, according to theWyss Institute. Gene drives can aid in controlling the spread of diseases such as malaria by enhancing sterility among the disease vector

Where it gets tricky is this technologys potential to enhance or eliminate certain traits of parents to their offspring; Im talking of course about designer babies.

Obviously, this is a discussion that extends well beyond the scope of this article. However, I will say this: the possibility of improving the human condition is likely worth the risk of unintended consequences. Therefore, I remain bullish on CRSP stock despite some nuances associated with the underlying narrative.

Matthew McCall left Wall Street toactually helpinvestors by getting them into the worlds biggest, most revolutionary trends BEFORE anyone else. The power of being first gave Matts readers the chance to bank +2,438% in (STMP), +1,523% inUltaBeauty (ULTA) and +1,044% in Tesla (TSLA), just to name a few.Click here to see what Matt has up his sleeve now. Matt does not directly own theaforementioned securities.

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3 Reasons to Go Long Crispr Therapeutics - InvestorPlace

GRIN Disorder, Kidney Failure and Fathers Day How One Family is Surviving it All –

My life changed forever the day I became the father to a son with extreme special needs. But I could never have imagined just how much being Brysons dad would continue to change my identity. While our familys COVID-19 isolation has been challenging, it has provided me with the opportunity to reflect on the evolving nature of my role as father.

Happy Faw-vers Day.

My two-and-a-half-year-old son Connor stumbles toward me, carrying a gift almost as big as he is. I unwrap a PlayStation 2. Its a surprisingly expensive gift, but my wife Laura reminds me that this is a special Fathers Day. Shes seven months pregnant, and even though we havent met our second child, were already in love with the newest member of our family.

I thank Connor before leaning in close and kissing Lauras belly. Thank you, baby.

If you'd like to make your Father's Day crafty, have your little ones try a fun printable print it here.

Baby Brysons first year has been tough. Hes missed all his milestones and doctors have confirmed that he isnt developing typically. Hes 10 months old but cant sit independently, or crawl, or track objects with his eyes. Hes nowhere near being able to say his first word or take his first steps. But doctors dont know why.

When Connor was born, my identity changed. I was no longer just a guy. I was a father. Becoming a special needs dad has changed me once again. The responsibility feels weightier; theres less room for error. I need to get this right.

A few days before Fathers Day, we go out for dinner at an all-you-can-eat Asian restaurant. The food is tasty but the service is slow. And Bryson is loud. Theres this sound he makes that we call singing but its really more of a happy wail. Were accustomed to it, but the diners around us are not. Someone complains and a manager drops by our table.

"You need to tell your son to be quiet," she says, waving at Bryson. "Other guests are complaining."

"My single greatest fear is whos going to protect him when Laura and I arent around."

We ask her to pack up our food and we head home, full of sadness and fear for Brysons future. Once hes in bed, I sit down and type out what I call my Fathers Day pledge, to be by his side to protect him from the judgments of this world until the day I die.

My single greatest fear is whos going to protect him when Laura and I arent around.

You can listen to Keith McArthur and Bryson's story in his podcast Unlocking Bryson's Brainright now. Listen here.

This ones hard. My own father died of cancer a couple months earlier. Theres this massive hole in my life that cant be filled, and on Fathers Day, it feels particularly unbearable. Ive become so depressed over the past couple of months that I cant seem to handle life anymore. I find myself leaving my desk at work and sneaking off to my car to hide, sometimes for hours.

Fears about Bryson are also weighing heavily. Hes almost eight but he cant walk or talk. His brain seems to be locked at the developmental age of 12 months. He also has violent episodes where he hurts himself and those around him. Hopes that he might just grow out of this are a distant memory. Doctors have performed so many tests dozens of blood draws, MRIs, muscle biopsies but they cant figure out whats causing Brysons condition.

I love Bryson so much, but I cant help wondering if hes even capable of loving back.

It all feels too hard. I promise Laura Ill see a doctor about my depression. Two weeks later Im on anti-depressants. Life isnt easy, but at least its manageable again.

Were celebrating Fathers Day in a hotel room in suburban Pittsburgh. Connor hands me some gifts, including a barbecue cookbook and a broken picture frame, the casualty of an overpacked suitcase and a six-hour road trip.

Our lives have changed dramatically over the past two years. I got off the anti-depressants, then experienced kidney failure, then got a second chance at life when my sister Stephanie donated one of her kidneys to me.

And after nearly a decade of searching, we finally got a diagnosis for Bryson. A genetic sequencing test revealed that Bryson has a mutation in his GRIN1 gene one of several genes that encode receptors in the brain that play a critical role in learning and memory. Pathogenic variants in this cluster of genes are known as GRIN Disorder.The diagnosis is why were here in Pittsburgh, meeting other GRIN Disorder families from across the country. Bryson is no longer alone.

Weve agreed to host this years GRIN family conference in Toronto, so I spend Fathers Day finalizing plans. Families travel in from across North America and we line up a panel of scientists who can help us understand this rare disease. Im shocked when they tell us that a cure for GRIN Disorder might be possible. Bryson is perfect; I dont want to change him. But if new treatments could allow him to communicate and make more of his own decisions, wouldnt he want that?

My paternal identity changes once again. Im no longer just a father or a special needs dad, but a rare disease warrior putting my life on hold to chase down a cure. I make a podcast series about this journey. Unlocking Brysons Brain launches two years later in the midst of a global pandemic.

The world is slowly getting back to normal but not for my family. GRIN Disorder usually isnt fatal, but some GRIN kids have died suddenly from respiratory complications. Im also on post-transplant drugs that lower my immune system. We cant risk allowing COVID-19 into our family, so well be spending this Fathers Day much as we have spent the past three months in isolation.

Brysons medical appointments are all on hold. So is the physio and speech therapy he usually gets through school. And the Grade 8 graduation ceremony Id been looking forward to for years? Its not happening this year. Worse still, all the research Ive been pushing to help find a cure is on hold as labs that arent focused on COVID-19 shut their doors.

On the plus side, pandemic life means more family time. And on a cloudy weekend in May, my son Bryson delights me with two early Fathers Day gifts.

Bryson does well in his walker at school, but hes been struggling at home. He just plops down on the safety seat instead of moving forward. So Laura has an idea. She removes the seat. And suddenly Bryson takes off, walking better than Ive ever seen him move before. Its the best gift I could hope for.

"We cant risk allowing COVID-19 into our family...."

But theres a bigger one yet to come.

Bryson loves it when I kiss him on the left side of the neck, just under his ear. When he wants me to do this he puts his hands on the back of my head and pulls me close. But this time, he pulls me in close and, for the first time, starts kissing my face. The kisses are wet and toothy, and make both of us erupt with laughter.

And they are unmistakable acts of love.

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GRIN Disorder, Kidney Failure and Fathers Day How One Family is Surviving it All -

Gene Therapy: Regal Profits From Curing "The Royal Disease"? – Sick Economics

A Cure for Hemophilia? BioMarin Attempts to Stop the Bleeding with a Single Shot

By Matthew Rojas, Biotech Analyst

Most people recall learning about hemophilia from the history books. Specifically, Queen Victoria of England, who ruled from 1837-1901, was a suspected carrier of the hemophilia B trait that causes the infamous, potentially fatal bleeding disorder. Since then, hemophilia has commonly been referred to as a royal disease due to its prevalence in various royal families throughout England, Germany, Russia, and Spain during the 19th and 20th centuries.

In the United States, there are approximately 20,000 people with hemophilia, and an estimated more than 400,000 individuals have the disorder worldwide. In addition to hemophilia B, there is also hemophilia A. The difference between the two conditions lies in which clotting protein is defective or missing for hemophilia B it is factor IX and for hemophilia A, factor VIII. Nevertheless, the symptoms of both variations of hemophilia are the same and range in severity from excessive bleeding only after an injury to frequent, spontaneous bleeding.

Presently, people with hemophilia A or B need infusions of the respective clotting protein two to three times per week. Not to mention, the weekly injections do not stop all bleeding episodes. Going to the doctor this frequently is extremely time-consuming, making it inconvenient for most individuals. Hence, many people with hemophilia fail to receive adequate treatment for their condition, which puts them at great risk. There is a pressing need for a more efficient way to treat individuals with hemophilia this is where BioMarin comes to the rescue.

BioMarin is a California-based biotechnology company that focuses on rare diseases driven by genetic causes. Currently, they have something revolutionary in their clinical pipeline for patients suffering from hemophilia A: valoctocogene roxaparvovec or valrox. Unlike traditional methods for treating hemophilia, valrox does not require repeated injections to temporarily mitigate symptoms; valrox uses gene therapy and only requires a single infusion to cure hemophilia A patients. Yes, you read that right cure. Gene therapy is a complicated process that works by inserting genetic material, via a carrier or vector, into cells so that they can make a protein in this case, factor VIII. For valrox, the vector is AAV5; a concern arose because people with AAV5 antibodies are ineligible to receive valrox. However, BioMarin estimates that 80% of people with hemophilia A do not have AAV5 immunity, so this should not be a problem.

A cure almost seems too good to be true, but the data are clear. In BioMarins Phase III clinical trial, seven out of 16 hemophilia A patients exhibited required levels of factor VIII. The company submitted this data along with three years of Phase I/II data for FDA approval. The application is currently under priority review, and a decision is expected on August 21, 2020. Therefore, now is the perfect time to invest in BioMarin because its current stock price does not reflect the potential revenue from valrox in the near future.

According to Hank Fuchs, president, Global Research and Development at BioMarin, Valoctocogene roxaparvovec has the potential to be the first gene therapy to be approved in any type of hemophilia. In the gene therapy space, it is critical to be a first mover because once patients are treated, they are taken off the market. After all, people only need a single shot.

Some of BioMarins competitors include Pfizer and Sangamo Therapeutics SB-525 and Spark Therapeutics SPK-8011, both gene therapies for hemophilia A. However, SB-525 is still in the process of Phase III clinical trials, and Spark ran into trouble when one patient ended up in the hospital due to an immune reaction related to SPK-8011. On the other hand, valrox is safe, has already completed its clinical trials, and is currently under priority review by the FDA. Thus, BioMarin is on track to be the first mover.

To further ensure its dominance in the hemophilia A gene therapy space, BioMarin increased the number of doses its gene therapy facility could produce from 4,000 to 10,000. To put that increase in perspective, the company can now treat all of the hemophilia A patients in the United States in two years. If established as the first mover, BioMarin has sufficient capacity to quickly dose hemophilia A patients before other gene therapies are approved to go to market.

As stated above, the current treatments for hemophilia A are weekly, expensive injections of prophylactic FVIII. Patients are dependent on these injections for their entire lives, costing an estimated $700,000 to $750,000 per year. Also, many hemophilia patients have greater costs due to frequent hospital visits, procedures, various tests, etc.

One study analyzes the possible cost-effectiveness of valrox compared to traditional prophylactic FVIII injections. The results are astounding the average patient cost of valrox is an estimated $16.7 million, whereas the average patient cost of the prophylactic FVIII injections is an estimated $23.5 million. Therefore, valrox provides a reduction in costs of $6.8 million per patient, on average. Because valrox is cheaper and more effective, it is likely to become the dominant way that hemophilia A patients are treated.

Unlike many biotechnology companies, BioMarins stock (BMRN) has come out on top at the tail-end of the COVID-19 pandemic. Before the crisis, the company was trading at around $97.00, and now the share price has increased by about 10% to $106.00. This increase in share price is promising because if BioMarin can perform well in times of economic-downturn, imagine what they can do in times of economic health. The reason for this continued growth is likely because BioMarin has steady revenue streams from a diversified portfolio of products for rare diseases; in 2019 alone, the company generated nearly $1.7 billion in revenue from its product lines, a 13% increase compared to 2018. With the likely approval of valrox in late August, I expect that the stock price will increase even further.

Furthermore, BioMarin has solid financials across all of its first-quarter 2020 statements. As of its most recent balance sheet, BioMarin has a current ratio slightly above 1, indicating that it can cover its short-term debt obligations. Additionally, the company generated over $100 million in revenues in the first quarter, a 25% increase from the fourth quarter of 2019. Lastly, BioMarin ended the most recent quarter with an increase of about $40 million in cash, which shows that it can generate liquidity even in times of crisis. BioMarins financials are stronger than ever, allowing it to continually achieve success.

Hundreds of thousands of hemophilia patients around the world have an unmet need: a permanent treatment for their disorder. BioMarin meets that need. With the use of revolutionary gene therapy, BioMarin has developed valrox, a single infusion that will cure hemophilia A patients once and for all. Not only is valrox more effective than the leading treatments for hemophilia A, but it is also much cheaper, saving patients and payers millions of dollars. With BioMarin scheduled to be the first-mover in the hemophilia A gene therapy space, now is the critical time to invest before valrox stops the bleeding.

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Gene Therapy: Regal Profits From Curing "The Royal Disease"? - Sick Economics

Cancer patients and doctors carry on with clinical trials during Covid-19 – STAT

Cancer isnt waiting for Covid-19 to go away. Neither is Karen Howley.

Diagnosed two years ago with advanced ovarian cancer, Howley started on a new experimental drug in mid-March, just as coronavirus case counts were soaring in Massachusetts. Her treatment, part of a clinical trial at Dana-Farber Cancer Institute in Boston, began with a four-week hospital stay, and since then, shes been returning every Monday for infusions.

While the study drug dripped into her body, she heard the hospital staff apprehensively discussing the coming surge of cases. Howley felt safe on a floor devoted to cancer patients, where there was no shortage of personal protective gear for the medical workers, but she still wiped down her tray each time a a meal was delivered to her bedside.


Ambulance sirens wailing endlessly outside her window made her anxious. The hospitals no-visitor policy made her lonely. But Howley, who has faced her cancer with both humor and realism, never considered quitting the clinical trial.

I dont think it occurred to me, Howley, 56, said during an infusion appointment last week that she wryly called her spa day.


Treatment for me is prolonging my life at this point. And if I do get Covid, I do.

Around the world, Covid-19 has disrupted all types of clinical trials, decreasing by 74% the number of new patients enrolling. In cancer, the number of new patients entering Phase 2 and 3 cancer trials in the U.S. plummeted by about 46% to 48% in the last two weeks of March, said Margaret Mooney, associate director of the Cancer Therapy Evaluation Program in the Division of Cancer Treatment and Diagnosis at the National Cancer Institute. By the last week of May and first week of June, the cliff wasnt so steep, moderating to a 25% to 30% decline.

Some medical centers put certain research trials on hold, stretched too thin by the needs of treating Covid-19 patients. Others continued their cancer trials by taking advantage of NCI-modified protocols allowing patient consent by phone or delivery of oral drugs to patients homes. Depending on where an individual site was located, they made local decisions that were best for their patients and their patient population in terms of clinical trials, Mooney said.

At Dana-Farber, Ursula Matulonis, chief of gynecologic oncology and Howleys oncologist, recalled patients feeling frightened and doctors feeling uncertain about exactly how they would carry on in the early days of the pandemic but no one ever doubted that they would.

We said, look, weve got to go in to see our patients because theyve got cancer. They want to continue to be treated in the same way, she said. We have a job to do and we have to continue to do that job.

Doing that job at Dana-Farber means patients are screened for symptoms when they enter the cancer center and at each step along the way. Howley answers the same set of questions Any fever? Any cough? Any contact with anyone who has Covid-19? when she has her vitals checked, gets her blood drawn, sees her doctor, receives her infusion.

Shes used to the waiting-room chairs zip-tied in opposite directions to keep single patients (no more friends or family) from getting too close. She expects to park her own car rather than turn it over to a valet. Because shes enrolled in a research trial, there are more blood samples taken for analysis and for tracking her response to the drug, but the entire visit moves along more quickly now, streamlined to reduce waiting times so no patient is exposed longer than necessary to other people.

Andrew Wagner, an oncologist who is leading Dana-Farbers efforts to keep patients safe and on treatment during Covid-19, said in consultation with study sponsors, some tissue biopsies were canceled, or for some patients chemotherapy was adjusted from every week to every three weeks. Physician visits to discuss imaging scans moved from in-person to video. Dana-Farber had zero telemedicine before March, but virtual visits now account for just under half of patient appointments, holding lessons for the future.

Its been hard in oncology, of course. Such a large part of what we do is the emotional care of patients and their families, Wagner said. Being able to hold someones hand is really hard to do through telemedicine, but certainly the video aspect of it is much better than just the telephone. Youre able to express yourself empathically.

Patient visits to Dana-Farber plunged by 40% in the course of one week in mid-March but chemotherapy appointments slipped by only 10% to 15% over that same week. Now doctors are encouraging patients to come back in. We think that its very safe here. We are certainly safer than in the grocery store, Wagner said. Cancer is not going to wait for Covid to go away.

Other cancer centers made similar adjustments. At the Ohio State University Comprehensive Cancer Center in Columbus, where close to 1,000 clinical trials are typically underway, Ohio Gov. Mike DeWines March 23 stay-at-home policy kept 200 research nurses and other staffers away from their duties. That meant only trials with a critical therapeutic outcome, extremely modest staffing needs, or time-sensitive enrollment targets continued, said Raphael Pollock, director of the cancer center.

Now that Ohios stay-at-home policy has been lifted and the research machinery is gearing up again, he expects trial participation to tick back up, too. Cancer didnt take time off, Pollock said. Well be quite busy by the mid- to late summer.

And at Memorial Sloan Kettering Cancer Center in New York City, the U.S. epicenter of the pandemic, no therapeutic trials were put on hold, but in-clinic volume in March and April did drop by about 75%. Telemedicine helped, said Paul Sabbatini, an oncologist and deputy physician-in-chief for clinical research. We and most centers have developed processes to rationally deal with the Covid risk as best we can so [clinical trials] can proceed now.

Now patients are coming back.

While it was prudent for patients to avoid the medical setting when the pandemic was at peak, enough time has elapsed that many of those patients clearly now have a benefit/risk ratio in favor of care, Sabbatini said. Clinical trials remain the only way we evaluate and ultimately approve new therapies for patients with cancer.

NCIs Mooney believes some good may come from adjustments made to ensure the safety of patients participating in studies and medical staff. But its still a work in progress, as hotspots rise and fall.

I think were learning more as we go along, she said. Some of the adaptations weve made have made all of us realize that perhaps there is better use we can make of technology telemedicine or technologies like that to take care of the patient. Thats a benefit to everyone, and they may be things that we can continue into the future once the public health emergency, as we all hope, has been resolved.

At Dana-Farber, Howley is enrolled in a Phase 1 trial of REGN4018, a bispecific antibody developed by the biotech company Regeneron. It grabs onto cancer cells at two points connected by a bridge. One side targets MUC16, a gene mutated in ovarian cancer and previously known as CA125 (which is still the name for the biomarker measured in diagnostic blood tests). The other side of the antibody binds to a T cell receptor that can then kill the cancer cell, the theory goes.

You realize this [Covid-19] is temporary and one day we will be back. For me, its like, I dont know how long I have.

When patients are hospitalized at the outset of the trial, which at this early phase is designed to test safety and dosing, its not an easy time for them with or without a pandemic. They are waiting for the drug to provoke their immune systems into killing their cancers. Howley felt savage pain across her abdomen and the wrath of a cytokine storm, the massive inflammatory response that also occurs in some Covid-19 patients whose immune systems go into overdrive.

We believe that the best antibodies you can get are the specific antibodies that travel to the sites of tumors. And thats what causes the abdominal pain, Matulonis, her oncologist, said.

From the beginning, Howleys treatment path has not been easy. After initial surgery to remove her tumor, she endured peritoneal delivery of the chemotherapy drug cisplatin directly to her abdomen, which was both painful and ultimately ineffective. She went on a standard drug called Doxil, but her cancer still progressed. Next she started a clinical trial that combined three drugs: one immunotherapy drug, one drug that blocks new blood vessel growth, and one PARP inhibitor that interferes with how cancer cells repair DNA damage. It was an aggressive regimen designed to expose the cancer to three agents it had never seen before, Matulonis said.

But one drug caused a bowel perforation, a known side effect. She continued to take the immunotherapy and the PARP inhibitor, but by November her cancer was progressing, measured by blood tests gauging her CA125 levels and imaging scans that spot metastasis.

The new trial, of the bispecific antibody, started in February.

I think for her, Ive always wanted to go beyond standard chemotherapy, Matulonis said. You can see that the tumor was a harder nut to crack. We really have to think outside the box to treat her.

Matulonis said Howley can stay on the current trial as long as she meets three conditions: She is benefiting from the drug, she is not having any significant toxicities, and she wants to continue. Howleys CA125 has hit four figures in the past. Before last weeks blood test, her last reading was 499. She was both nervous and eager to know the latest, though she tries not to put too much stock in the biomarker. The reading can vary with inflammation and the drug she is on causes inflammation, irritating one hip so much that she wants to ask if a cortisone shot would be allowed under the trials rules.

Except for the cancer, shell tell you shes healthy, and Matulonis agrees. Retired now from a career in client services, she lives with her husband in Sudbury, Mass., about a 40-minute drive to Boston. She walks three miles every morning, and she just got back to playing tennis. She ran the Falmouth Road Race last summer, astonishing a research nurse on her medical team who was volunteering on the sidelines.

For her visit last week, she wore a white, cowl-necked knit top port-friendly for blood draws and drug infusions through an opening high on her chest. Her lively blue eyes were set off by sparkly deep-blue eye shadow, her dark hair accented with blond highlights. Shes been the woman in the waiting room with no hair or eyebrows and knows how it feels when people look at you. Now she jokes about reluctantly giving up her fashion-coordinated cloth face mask for the fresh paper one Dana-Farber offers each patient upon check-in.

Howley has dual motivations for participating in cancer research. One is to help other women with ovarian cancer. Hopefully Im a piece of that puzzle thats going to give them a little insight, she said. They might have something here thats going to be so critical down the road for others.

Her other driving force is her 29-year-old daughter. She doesnt worry about their sharing BRCA1 or BRCA2 mutations her genetic tests were negative for those mutations but she is concerned about being a role model.

So much of my determination is just really providing an example to her, she said. Im not saying Ive got it down at all, but I try very hard to show her how to do it kind of gracefully. It doesnt mean Im going to do it. But I still try.

Cancer has already narrowed how she thinks about the future. Six months is as far out as shell plan these days, and Covid-19 has cramped her dreams of traveling to one of her favorite places, Marco Island in Florida. You realize this [Covid-19] is temporary and one day we will be back, Howley said. For me, its like, I dont know how long I have.

No one can tell her that, but she looks to Matulonis to see if her new CA125 number matches how good shes felt, even if she hit lousy on the tennis court.

The number is still moving in the right direction, Matulonis tells her: 259, down from 499 a month ago. That could mean her cancer burden is diminishing, Matulonis said, but cautioned that imaging scans will have to confirm it.

Howley knows that.

If it goes up 50 points, it doesnt necessarily mean anything. But at the same time, Im just blown away, she said. Today, its almost surreal to me, its going to take me a while to digest.

She is still elated as she climbs into the infusion chair for her next dose of the trial drug.

Maybe I better plan that trip.

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Cancer patients and doctors carry on with clinical trials during Covid-19 - STAT

Genentech’s Tecentriq in Combination With Chemotherapy (including Abraxane) Meets Primary Endpoint of Improved Pathological Complete Response,…

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced that the Phase III IMpassion031 study, evaluating Tecentriq (atezolizumab) in combination with chemotherapy (Abraxane [albumin-bound paclitaxel, nab-paclitaxel]; followed by doxorubicin and cyclophosphamide) in comparison to placebo plus chemotherapy (including Abraxane), met its primary endpoint by demonstrating a statistically significant and clinically meaningful improvement in pathological complete response (pCR) for the treatment of people with early triple-negative breast cancer (TNBC), regardless of PD-L1 expression.

Triple-negative breast cancer remains an aggressive disease with high rates of recurrence, said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. Our goal in treating TNBC at its earliest stages is to provide people with the best chance for a future cure. Adding Tecentriq to chemotherapy now has the potential to help women with TNBC at multiple different stages of the disease.

In the study, fewer patients who received the Tecentriq combination as a neoadjuvant (before surgery) treatment had evidence of tumor tissue detectable at the time of surgery (known as pCR), regardless of PD-L1 expression, in comparison to the control arm. Neoadjuvant treatment may allow a doctor to quickly assess whether a medicine is working, and may also reduce a tumor's size so it is easier to surgically remove. pCR is a common measure of neoadjuvant treatment effect in breast cancer and can be assessed more quickly than traditional endpoints in early-stage breast cancer.

Safety for the Tecentriq combination appeared to be consistent with the known safety profiles of the individual medicines and no new safety signals were identified. Results of the IMpassion031 study will be presented at an upcoming medical meeting and will be discussed with global health authorities including the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA).

The IMpassion031 study is the second positive Phase III study from Genentech demonstrating the benefit of Tecentriq in TNBC, and the first Tecentriq study to demonstrate benefit in early TNBC. Tecentriq in combination with nab-paclitaxel is currently approved in more than 70 countries worldwide, including the U.S. and across Europe, for the treatment of adults with unresectable locally advanced or metastatic TNBC in people whose tumors express PD-L1 (IC1%).

Genentech has an extensive development program for Tecentriq, including multiple ongoing and planned Phase III studies across lung, genitourinary, skin, breast, gastrointestinal, gynecological, and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines.

About the IMpassion031 study

The IMpassion031 study is a Phase III, multi-center, randomized, double-blind study evaluating the efficacy and safety of Tecentriq (atezolizumab) in combination with chemotherapy (Abraxane, [albumin-bound paclitaxel; nab-paclitaxel]; followed by doxorubicin and cyclophosphamide) in comparison to placebo plus chemotherapy, in people with previously untreated, early TNBC. The primary endpoint is pCR using the American Joint Committee on Cancer (AJCC) staging system in the intention-to-treat (ITT) population and in the PD-L1-positive population. Secondary endpoints include overall survival, event-free survival, disease-free survival and quality of life measures.

About Tecentriq (atezolizumab)

Tecentriq is a monoclonal antibody designed to bind with a protein called PD-L1. Tecentriq is designed to bind to PD-L1 expressed on tumor cells and tumor-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the re-activation of T cells. Tecentriq may also affect normal cells.

About triple-negative breast cancer

Breast cancer is the most common cancer among women worldwide. According to the American Cancer Society, close to 280,000 people in the United States will be diagnosed with invasive breast cancer, and more than 42,000 will die from the disease in 2020. Breast cancer is not one, but many diseases based on the biology of each tumor. In triple-negative breast cancer, tumor cells lack hormone receptors and do not have excess HER2 protein. Approximately 15 percent of breast cancers are triple-negative based on the results of diagnostic tests. It is an aggressive form of the disease with few treatment options.

Tecentriq U.S. Indications (pronounced t-SEN-trik)

Tecentriq is a prescription medicine used to treat adults with:

A type of bladder and urinary tract cancer called urothelial carcinoma.

Tecentriq may be used in patients with urothelial carcinoma if their bladder cancer has spread or cannot be removed by surgery, and if they have any one of the following conditions:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called non-small cell lung cancer (NSCLC).

Tecentriq may be used alone as the first treatment in patients with lung cancer if:

Tecentriq may be used with the medicines bevacizumab, paclitaxel, and carboplatin as the first treatment in patients with lung cancer if:

Tecentriq may be used with the medicines paclitaxel protein-bound and carboplatin as the first treatment in patients with lung cancer if:

Tecentriq may be used alone in patients with lung cancer if:

If a patients tumor has an abnormal EGFR or ALK gene, they should have also tried an FDA-approved therapy for tumors with these abnormal genes, and it did not work or is no longer working.

A type of breast cancer called triple-negative breast cancer (TNBC).

Tecentriq may be used with the medicine paclitaxel protein-bound in patients with TNBC when their breast cancer:

The approval of Tecentriq in these patients is based on a study that measured the amount of time until patients disease worsened. Continued approval for this use may depend on the results of an ongoing study to confirm benefit.

A type of lung cancer called small cell lung cancer (SCLC).

A type of liver cancer called hepatocellular carcinoma (HCC).

It is not known if Tecentriq is safe and effective in children.

Important Safety Information

The most important information about Tecentriq is:

Tecentriq can cause the immune system to attack normal organs and tissues and can affect the way they work. These problems can sometimes become serious or life-threatening and can lead to death.

Patients should call or see their healthcare provider right away if they get any symptoms of the following problems or these symptoms get worse.

Tecentriq can cause serious side effects, including:

Getting medical treatment right away may help keep these problems from becoming more serious. A healthcare provider may treat patients with corticosteroid or hormone replacement medicines. A healthcare provider may delay or completely stop treatment with Tecentriq if patients have severe side effects.

Before receiving Tecentriq, patients should tell their healthcare provider about all of their medical conditions, including if they:

Patients should tell their healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

The most common side effects of Tecentriq when used alone include:

The most common side effects of Tecentriq when used in lung cancer with other anti-cancer medicines include:

The most common side effects of Tecentriq when used in TNBC with paclitaxel protein-bound include:

The most common side effects of Tecentriq when used in hepatocellular carcinoma with bevacizumab include:

Tecentriq may cause fertility problems in females, which may affect their ability to have children. Patients should talk to their healthcare provider if they have concerns about fertility.

These are not all the possible side effects of Tecentriq. Patients should ask their healthcare provider or pharmacist for more information. Patients should call their doctor for medical advice about side effects of Tecentriq.

Report side effects to the FDA at (800) FDA-1088 or Report side effects to Genentech at (888) 835-2555.

Please visit for the Full Tecentriq Prescribing Information for additional Important Safety Information.

About Genentech in cancer immunotherapy

Genentech has been developing medicines to redefine treatment in oncology for more than 35 years, and today, realizing the full potential of cancer immunotherapy is a major area of focus. With more than 20 immunotherapy molecules in development, Genentech is investigating the potential benefits of immunotherapy alone, and in combination with various chemotherapies, targeted therapies and other immunotherapies with the goal of providing each person with a treatment tailored to harness their own unique immune system.

In addition to Genentechs approved PD-L1 checkpoint inhibitor, the companys broad cancer immunotherapy pipeline includes other checkpoint inhibitors, individualized neoantigen therapies and T cell bispecific antibodies. For more information visit

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit

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They discover how to stop tumor growth in breast cancer – Explica

A new study by researchers at Tulane University, located in the city of New Orleans, United States, revealed the discovery of the gene behind the most aggressive form of breast cancer, as well as how to disable it to curb tumor growth and cancer spread.

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The report published in the Scientific Report of the journal Nature, explains that the research focused on triple negative breast cancer (TNBC), the most aggressive form of this type of cancer. It is a difficult disease to treat and where the chances of survival are lower than in other types of this cancer.

The researchers looked at the role two specific genes play in TNBC called Rab27a and TRAF31P2. Subsequently, they suppressed these two genes causing them to stop working and found positive impacts in the fight against breast cancer.

Doctor Reza Izadpanah, team leader, explains:

Our findings show that both genes play a role in the growth and metastasis of breast cancer. Although the Rab27a target slows the progression of tumor growth, it does not affect the spread of small numbers of cancer cells or micrometastases. Conversely, attacking TRAF3IP2 suppresses tumor growth and spread, and interfering with it reduces preformed tumors and prevents further spread. This exciting discovery has revealed that TRAF3IP2 may play a role as a novel therapeutic target in the treatment of breast cancer.

And it is that the scientists of the University of Tulane discovered that when they turned off the TRAF3IP2 gene, the metastasis of the cancer, that is, its spread, did not take place for a full year after treatment. Furthermore, deletion of this gene was also related to stagnant tumor growth and shrinking the tumor to undetectable levels.

So far, these positive effects have been seen in animals, meaning that gene deletion therapy has yet to be tested in humans. However, Izadpanah and his team are quite positive about it and have already turned to the FDA. to speed up the path and be able to carry out clinical trials.

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They discover how to stop tumor growth in breast cancer - Explica

The subtle signs that point the way towards haemochromatosis – The Irish Times

The first symptom I had was a hot flush. To say peri-menopausal was not on my radar is an understatement says Cathy Brooks, development manager with the UCD Foundation.

I had no idea. Id just lost my mum in October and I remember by Christmas being with friends in Collins Barracks, having a coffee walking around and I just started getting these hot flushes and I was thinking what the hell is this? To be honest, I just attributed it to the grief and losing my mum and all of that.

It was only when I googled it and I saw the symptoms that I thought, could I be? Im too young.

She was 39 at the time.

Initial blood tests suggested Cathy was not peri-menopausal but with her symptoms continuing she decided to seek advice from a second GP. On hearing her symptoms, the doctor immediately suspected haemochromatosis and decided to carry out a blood test.

I like to think Ive a very positive outlook on life, Cathy says, explaining how she made the decision that she wasnt going to focus on this. I just got on with living life.

Every year I go for a check-up in the haematology clinic in St Vincents . They do your bloods before and they check your liver, your heart all the organs that can be damaged. Its like going in for an annual MOT really, she continues.

Only in the last six months Ive started to get really achey in my index finger and my middle finger. When I close my fist I can feel my fingers kind of crunching. Thats a symptom of haemochromatosis.

Because Cathys joints are getting really stiff, she has been referred to a rheumatologist.

Cathy describes herself as being very active.

I do a lot of sport. I do it for my mental health as well as my physical health, she adds, discussing her love of rowing and cycling. But this year, like most of the world, Ive just been sitting on the couch eating, she says. Im more stiff now because Im not active.

Cathy has phlebotomies every three months to treat her haemochromatosis but shes keen that her blood doesnt go to waste just because of her condition.

If Ive to give blood for the rest of my life, I dont want my blood to be dumped because theres too much iron in it. I want my blood used. So I go to this clinic where they can use my blood, she says adding that people with haemochromatosis travel from all over the country to the clinic in Stillorgan for this very reason.

The service has been extended and now, many people with haemochromatosis can become regular blood donors at all blood donation clinics nationwide, including local community clinics.

Potential donors must meet specific haemochromatosis management criteria, the Irish Blood Transfusion Service states, which includes having completed iron depletion therapy, and not suffering from any serious complications as a result of haemochromatosis.

They must also satisfy standard blood donation criteria to be able to attend clinics to give blood which can then be used to save patients lives throughout the country

Thankfully, Cathy hasnt experienced any difficulties giving blood in Covid-19 times.

I happened to give blood about two weeks ago. Ive heard loads of people are having trouble giving blood during coronavirus. I havent had any problem. Ive had nothing but a positive experience with all of this and all the professionals.

What is haemochromatosis?

Haemochromatosis is a genetic condition which, in many people who have the condition, gives rise to excess iron absorption and retention within the body, explains Prof Suzanne Norris, professor of hepatology and gastroenterology, Trinity College, Dublin.

Over time, and its a very slow, gradual process, iron accumulation builds up and the iron is absorbed and deposited in various organs. We have the highest rate of haemochromatosis of any European and worldwide country

What are the symptoms of haemochromatosis?

Chronic fatigue is the main symptom, Prof Norris explains. Achey joints would be another. The problem with haemochromatosis is that it doesnt really have a symptom that would make you say aha thats haemochromatosis. Its all subtle signs. Its a disease thats under the radar because it doesnt produce a symptom that would make a doctor think that has to be that condition. Its literally chronic fatigue and unfortunately most chronic conditions will give rise to chronic fatigue.

How is haemochromatosis diagnosed?

To make the diagnosis, you need to have inherited the gene for excessive iron accumulation from both parents. Only about 50 per cent of people who have the two genes actually ever develop iron overload, so theres obviously another trigger, which may be environmental, we dont quite know, Professor Norris says.

What are the potential complications of haemochromatosis?

Typically the organs that end up having too much iron absorbed within them are the liver, the pancreas which may give rise to diabetes, the joints which may give rise to arthritis, Prof Norris explains.

Bronze diabetes, or brown discolouration of the skin is actually quite rare. There are other symptoms which may occur around depression and low mood. Its not clear whether thats just in relation to general inflammation because of the iron burden, or because of not feeling well and low mood associated with chronic ill health in that regard.

What is the treatment for haemochromatosis?

The treatment is very simple. Its blood letting or having blood removed on initially maybe a weekly or monthly basis in the acute stage when the person is coming in with a new diagnosis, ProfNorris explains.

Once the excess iron has been removed its like giving blood to the blood bank, you donate blood once or twice a year, that might be sufficient for most people. Some people may do it four times a year and that keeps the condition totally under control.

When the iron level is back within the normal range and its maintained at that level by either once to four times a year, depending on the persons needs, it keeps it completely under control and there is no risk of developing serious potential complications from late diagnosis once its diagnosed early, Professor Norris emphasises.

Our message is always, at this time of year, for healthcare professionals to really think about chronic fatigue could it be iron overload, rather than anaemia giving rise to chronic fatigue.

For more information, visit the Irish Haemochromatosis Association at (to donate 4, text IRON to 50300).

Follow this link:
The subtle signs that point the way towards haemochromatosis - The Irish Times

Coach and Little Grace: Mother of child with Down Syndrome gets life-changing phone call – WVTM13

Eighteen years ago, former Alabama football coach Gene Stallings made a difference in the life of a grieving woman and her beautiful newborn.It's a story about love and compassion, a story that has not been told until now.This is the story of The Coach and Little Grace:Her heart was heavy on Mother's Day 2002.Laura Davis was depressed and confused as her first child slept at her side.Was she crazy to feel so sad? So hopeless? After all, her daughter Grace was a mere 14 days old. Laura Davis was supposed to be happy.As Grace slept, Laura asked God for answers. How was she supposed to care for a child with Down Syndrome? How was she supposed to see the future as her child had just been diagnosed with two holes in her heart? How was she supposed to have hope when her doctor told her that little Grace would never walk, talk or read?Her husband was doing his residency in Columbus, Ohio, and while Chris was working hard, Laura cried. She was overwhelmed. She missed her hometown of Vestavia Hills. She missed talking about life and Bama football with her friends.And getting out of bed? It just wasn't happening.The phone rang, and Laura took a deep breath, inhaling tears that had fallen from her eyes."Hello," said Laura."Hello, Laura, this is Gene Stallings. I wanted to call to wish you a Happy Mother's Day."Laura's heart jumped.Was it really THE Gene Stallings? The head coach of her beloved Crimson Tide?Why in the world was Gene Stallings calling Laura Davis, who was in Columbus, Ohio?"I also want to congratulate you on your newborn, Grace," the Tide coach said.How in the world did Coach Stallings know about Grace?The two exchanged pleasantries, and then Stallings revealed why he was really calling: To give Laura Davis hope. To get her out of bed.The coach proposed a question to Laura:"What is the one thing that you want for little Grace? You want her to someday go to heaven, right?""Of course," Laura replied.And then came just one of many bits of advice that the coach offered up:"Well, Laura, Grace has a one-way ticket because God loves her and so do you," he said. "You may not know this right now, but you are in for the ride of your life. You will never stop loving little Grace."And another piece of advice from the coach:"If the Lord asked me if I would rather have had John Mark the way he is or had him as a 'normal' child, I would tell Him that I would rather have had Johnny just the way he is."Coach Stallings said "goodbye" and hung up the phone.The tears in Laura's eyes returned. But this time they were happy tears. She had chill bumps.Little Grace remained next to her, asleep.We never know when that moment might come, but on Mother's Day 2002 that moment came for Laura Davis.She closed her eyes and prayed, promising God that she was going to make the most of Grace's life and offer her daughter every ounce of love that she could muster.And suddenly, good things started to happen:The two holes in Grace's heart closed on their own.Grace smiled.Physical therapy began, then speech therapy.Laura and Chris would move back to Vestavia Hills as Grace turned two. Years of hard work followed. Reading, writing, speaking.Vestavia Hills' special education teacher Jennifer Greer came into their lives and Grace blossomed as a cheerleader, a student and a friend.God was good.And now for the cherry on top:Three days ago, Grace Davis, the girl who was doubted, mocked, and given little chance to live a fulfilling life, graduated from Vestavia Hills High School. Grace will enter Auburn's "Eagles" program this fall. Yes, 18 years after doctors doubted her, Grace Davis will be going to college.Chris and Laura Davis are enjoying life in Vestavia Hills. Chris is an ENT doctor, and their sons Jack and Will are growing fast.Laura tells me that she will never forget Mother's Day 2002, the day that she received an unexpected phone call not from a football legend, but rather from the father of a Down Syndrome child.It was a phone call that motivated Laura to get of bed and look to the future one that ended up being more amazing than she could have ever imagined.And now you know the story of The Coach and Little Grace.A story with a happy ending!

Eighteen years ago, former Alabama football coach Gene Stallings made a difference in the life of a grieving woman and her beautiful newborn.

It's a story about love and compassion, a story that has not been told until now.

This is the story of The Coach and Little Grace:

Her heart was heavy on Mother's Day 2002.

Laura Davis was depressed and confused as her first child slept at her side.Was she crazy to feel so sad? So hopeless? After all, her daughter Grace was a mere 14 days old. Laura Davis was supposed to be happy.

As Grace slept, Laura asked God for answers. How was she supposed to care for a child with Down Syndrome? How was she supposed to see the future as her child had just been diagnosed with two holes in her heart? How was she supposed to have hope when her doctor told her that little Grace would never walk, talk or read?

Her husband was doing his residency in Columbus, Ohio, and while Chris was working hard, Laura cried. She was overwhelmed. She missed her hometown of Vestavia Hills. She missed talking about life and Bama football with her friends.

And getting out of bed? It just wasn't happening.

The phone rang, and Laura took a deep breath, inhaling tears that had fallen from her eyes.

"Hello," said Laura.

"Hello, Laura, this is Gene Stallings. I wanted to call to wish you a Happy Mother's Day."

Laura's heart jumped.

Was it really THE Gene Stallings? The head coach of her beloved Crimson Tide?

Why in the world was Gene Stallings calling Laura Davis, who was in Columbus, Ohio?

"I also want to congratulate you on your newborn, Grace," the Tide coach said.

How in the world did Coach Stallings know about Grace?

The two exchanged pleasantries, and then Stallings revealed why he was really calling: To give Laura Davis hope. To get her out of bed.

The coach proposed a question to Laura:

"What is the one thing that you want for little Grace? You want her to someday go to heaven, right?"

"Of course," Laura replied.

And then came just one of many bits of advice that the coach offered up:

"Well, Laura, Grace has a one-way ticket because God loves her and so do you," he said. "You may not know this right now, but you are in for the ride of your life. You will never stop loving little Grace."

And another piece of advice from the coach:

"If the Lord asked me if I would rather have had John Mark the way he is or had him as a 'normal' child, I would tell Him that I would rather have had Johnny just the way he is."

Coach Stallings said "goodbye" and hung up the phone.

The tears in Laura's eyes returned. But this time they were happy tears. She had chill bumps.

Little Grace remained next to her, asleep.

We never know when that moment might come, but on Mother's Day 2002 that moment came for Laura Davis.

She closed her eyes and prayed, promising God that she was going to make the most of Grace's life and offer her daughter every ounce of love that she could muster.

And suddenly, good things started to happen:

The two holes in Grace's heart closed on their own.

Grace smiled.

Physical therapy began, then speech therapy.

Laura and Chris would move back to Vestavia Hills as Grace turned two. Years of hard work followed. Reading, writing, speaking.

Vestavia Hills' special education teacher Jennifer Greer came into their lives and Grace blossomed as a cheerleader, a student and a friend.

God was good.

And now for the cherry on top:

Three days ago, Grace Davis, the girl who was doubted, mocked, and given little chance to live a fulfilling life, graduated from Vestavia Hills High School.

Vestavia Hills City Schools

Grace will enter Auburn's "Eagles" program this fall. Yes, 18 years after doctors doubted her, Grace Davis will be going to college.

Chris and Laura Davis are enjoying life in Vestavia Hills. Chris is an ENT doctor, and their sons Jack and Will are growing fast.

Laura tells me that she will never forget Mother's Day 2002, the day that she received an unexpected phone call not from a football legend, but rather from the father of a Down Syndrome child.

It was a phone call that motivated Laura to get of bed and look to the future one that ended up being more amazing than she could have ever imagined.

And now you know the story of The Coach and Little Grace.

A story with a happy ending!

See more here:
Coach and Little Grace: Mother of child with Down Syndrome gets life-changing phone call - WVTM13

The Rundown: May 22, 2020 – Multiversity Comics

Welcome back to The Rundown, our daily breakdown on comic news stories we missed from the previous day. Have a link to share? Email our team at

In case you missed it, we got an exclusive preview of Sera and the Royal Stars #7.

Nathan W. Pyles Strange Planet will be getting a subscription box from Culturefly, The Pop Insider reports. Fans will be able to get exclusive merchandise inspired by the comic as part of this new subscription box. Each box includes a vinyl figure, an exclusive comic on a canvas print, and five to seven additional items featuring the creatures. The subscription box will launch on June 1.

Via Bleeding Cool, Doctor Who fans were treated to a special comic strip written by James Peaty, illustrated by Mike Collins, and narrated by Richard Ashton, in which fans find out more about Danny Pink. Its unclear when the next series of Doctor Who will premiere, although the show will return in the next year with the special Revolution of the Daleks.

Variety reports that Nick Jonas and Lawrence Fishburne are set to star in AGC Studios The Blacksmith, based on the graphic novel from Kickstart Comics by Malik Evans and Richard Sparkman. Pierre Morel (Taken) will direct from a screenplay adapted by Ben Ripley (Flatliners). Jonas will play Wes Loomis, a go-to weapons expert for the intelligence community, who goes on the run after his lab is destroyed and colleagues are murdered. Fishburne will play Mather, a retired blacksmith, and Loomiss mentor. With the aid of a CIA analyst named Noelle Hazlitt, the trio will embark on a journey that keeps this improbable pair one step ahead of their pursuers in a breathless, action-filled thriller. Production on the film will begin later in 2020.

Via, Cartoon Network announced that We Bare Bears: The Movie will officially release for purchase on digital platforms on June 8, 2020. This is the first movie for the We Bare Bears franchise, which follows three talking bears as they explore life in the woods and city. In addition to the release date, Cartoon Network has also revealed a poster and trailer. The movie will depict the bears as they make their way to Canada after being chased from their home by antagonist, Agent Trout. Eric Edelstein, Demetri Martin, and Bobby Moynihan star in the movie as the bears Grizz, Ice, and Panda. Agent Trout is voiced by Marc Evan Jackson and many of the shows guest stars will also be featured in the movie. We Bare Bears: The Movie is scheduled to release for purchase on digital platforms for $14.99 on June 8, 2020.

Netflix has released the official trailer for The Old Guard, based on the comic by Greg Rucka and Leandro Fernandez. Like the comic, the movie follows a group of immortal soldiers led by Andy, played by Charlize Theron, who must fight to keep their immortality a secret, or otherwise see their gifts exploited. The film will begin streaming on Netflix on July 10, 2020. You can check out the trailer here.

Deadline reports that Sony has hired Marc Guggenheim to write a script for Jackpot, another installment in the larger Spider-Man Cinematic universe following the Venom franchise, and Jared Letos Morbius. In the comics, Jackpot is Sara Ehret, a scientist, who, while pregnant, is doing gene therapy research and is exposed to Lot 777, a virus that rewrites the DNA in her cells. She becomes imbued with superhuman strength and takes on the name Jackpot. Morbius is scheduled to be released on March 19, 2021, while Venom: Let There Be Carnage is set to hit theaters June 25, 2021.

TV Line reports that ABC has renewed 13 shows on their current lineup, including Stumptown. The TV show is based on the comic book series from creators Greg Rucka and Matthew Southworth, and follows Dex, an army vet turned private detective. Season 1 premiered in September. Its unknown when production will begin on season 2 of Stumptown.

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The Rundown: May 22, 2020 - Multiversity Comics